WHO Information Vol. 31, No. 3, 2017 Regulatory news

Regulatory news

Pre-market assessment of adverse events in relation to stopping a trial or progressing to the next dosing level. Strategic approach to development ►► EMA Press release, 25 July 2017. of children’s medicine European Union, United States of America – The EMA and the U.S. FDA have finalized Generic products in the U.S. their joint proposal to promote the use of United States of America – The FDA has innovative approaches in the development taken two steps to expedite market entry of of medicines for Gaucher disease. The needed generic medicines. These actions are proposed strategies can apply to medicines among the first taken under the Agency’s development for rare diseases in children in Drug Competition Action Plan announced general. in late May. The strategy document encourages Firstly, to encourage generic drug medicine developers to make better use development, the FDA posted a list of of extrapolation of available clinical data branded that have no listed patents or from adults to children through modelling exclusivities and for which the agency has and simulation, and to conduct multi-arm, yet to approve an Abbreviated New Drug multi-company clinical trials on several Application (ANDA). The Agency intends new medicines at the same time, with the to expedite the review of generics on this same control arm serving more than one list, which will be refined and updated medicine under evaluation. The overall periodically. aim is to reduce the number of patients in Secondly, the FDA has announced a clinical trials while maintaining high quality change to its policy on prioritizing the standards, thus reducing the burden on review of needed generics. The review children and their families. of applications for a given medicine will ►► EMA News, 3 July 2017. be expedited until there are three FDA- approved generics for that medicine. This policy change is based on data indicating Revised EMA clinical trial guidelines that significant price reductions occur when European Union – The EMA has released its there are multiple FDA-approved generics revised guidelines for first-in-human clinical available. trials. The revision takes into account the ►► FDA News release, 27 June 2017. increasing complexity of trial protocols. It provides guidance on the calculation of the starting dose, subsequent dose escalations Joint EU assessment platform and criteria for the maximum dose. European Union – The EMA and the Guidance is also provided on criteria to stop European Network for Health Technology a study, the rolling review of emerging data Assessment (EUnetHTA) have launched especially regarding safety, and the handling a new joint platform that will facilitate

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alignment of data requirements with The sample panel consists of plasma evidence being generated for both samples from anonymous individuals regulators as well as the bodies that provide infected with Zika, West Nile, or dengue recommendations to payers and other viruses. Although the panel is not for decision-makers. research purposes, diagnostic developers The platform will enable medicine can use these samples to assess whether their developers to obtain simultaneous, tests can help distinguish recent Zika virus coordinated advice and health technology infection from infection with West Nile or assessment bodies. Patient representatives dengue viruses. Using the same serological will be involved in parallel consultations on panel to evaluate different devices available a routine basis. The improved consultation, under Emergency Use Authorization (EUA) coordination and streamlined logistics are will help public health professionals expected to lead to more robust outcomes. compare the performance of different Zika ►► EMA Press release, 4 July 2017. virus tests. The FDA panel is available to developers who have interacted with the Agency through the pre-EUA process and Emergency importation list have devices that are in the final stages of Canada – Health Canada has published an validation. Other developers interested in initial list of medicines for which there is requesting a panel may contact the Agency. an urgent public health need, and which ►► FDA News release, 17 August 2017. are authorized for sale in the U.S., the EU or Switzerland, but not yet in Canada. Health Canada will permit these drugs to be Post-market monitoring imported for use in Canada. Provisions are in place for reporting of adverse reactions EMA platform gains trade mark and organizing recalls. European Union – The EU Intellectual The initial list includes medicines to Property Office (EUIPO) has approved the treat opioid use disorder and tuberculosis. registration of the name “EU PAS Register” Medicines will remain on the list for one as a European Union trade mark. year, renewable if there is a continued need The EU electronic Register of Post- for access. Additional medicines may be Authorisation Studies (EU PAS Register) was added to the list in the future, for example to developed through the European Network treat pandemic viruses or to address other of Centres for Pharmacoepidemiology public and military health emergencies. and Pharmacovigilance (ENCePP), which ►► Health Canada News release, 28 June 2017. is coordinated by EMA. Launched in November 2010, this openly accessible platform currently includes information Standardized testing panel for Zika on more than 1 100 observational post- United States of America – The FDA has authorization studies, of which about a third made available a panel of human plasma are finalized. The trade mark will reinforce samples to aid in the regulatory evaluation of the EMA’s legal control over the name of the serological tests to detect recent Zika virus platform and its content. infection. ►► EMA News, 7 August 2017. EU PAS Register®

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Automated FDA field alert reports reports had been issued in July 2015, and United States of America – After the CDSCO had provided companies with a successful completion of a four-year pilot checklist of GMP and GLP requirements as phase, the FDA has released a new version specified under Schedule M and Schedule of its automated Form FDA 3331a for L-1 of the Drugs and Cosmetics Act and electronic submission of field alert reports Rules of India. However, CDSCO is yet for pharmaceutical products. The new to receive self-inspection reports from form does not require signatures, requires manufacturers. no additional software or licenses beyond ►► CDSCO Notice, 9 August 2017. Adobe Acrobat Reader and an email client, and enables the FDA to import data directly to its systems. Collaboration Field alert reports enable the regulators to quickly identify quality defects in distributed China Food and Drug products that may present a potential Administration joins ICH safety threat. The Agency is working on Montreal – At its meeting held in Montreal, the technical requirements for receiving Canada on 27 May to 1 June 2017, field alert reports as part of the electronic the Assembly of the The International Common Technical Document (eCTD) Council for Harmonisation of Technical through the electronic submissions gateway. Requirements for Pharmaceuticals for ►► FDA Notice to Industry, 15 June 2017. Human Use (ICH) approved the China Food and Drug Administration (CFDA) as a new Regulatory Member, and the Pharmaceutical GMP compliance Inspection Co-operation Scheme (PIC/S) as a new Observer. With these new parties, Indian manufacturers to submit ICH now has 14 members and 23 observers. self-certification Full details are available on the ICH website1. India – The Drugs Controller General At the meeting the ICH Assembly of India has issued a notice requesting also agreed to begin work on two new pharmaceutical manufacturers to submit topics: a harmonized guideline on the use their self-assessment reports and self- of extrapolation in children’s medicine certification of compliance with good development, and revised general manufacturing practice (GMP) and good considerations for clinical trials. The laboratory practice (GLP) requirements Assembly further adopted new guidance to the State Licensing Authorities and documents and made some revisions to to the Central Drugs Standard Control its Articles of Association and rules of Organization (CDSCO) by 30 August 2017. procedure to keep operations streamlined The notice states that issues related to the with a growing number of members and possibility of self-certification, followed by observers. third-party certification and detailed audit, ►► ICH Press release, 19 June 2017. have been deliberated at the highest level. CFDA News, 20 June 2017. An earlier notice requesting mandatory self-audits and submission of self-assessment

1 http://www.ich.org/about/membership.html

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U.S.-EU cooperation in inspections consolidated management committee is The European Commission (EC), the U.S. planned for June 2018. FDA and the EMA have signed a new The agreement follows an in-depth confidentiality commitment that allows review of various governance models. the US regulator to share non-public and The consolidation is expected to realize commercially confidential information several opportunities, including: enabling – including trade secret information a shared vision for information exchange relating to medicine inspections – with and regulatory cooperation; maximising EU regulators. This confidentiality synergies and avoiding duplication of commitment is a milestone in the ongoing effort; creating a regulatory hub for implementation of the mutual recognition of pharmaceuticals that covers all medicinal inspections of medicine manufacturing sites. products, enabling closer linkages with The EU and the U.S. have had initiatives to simplify the numerous forms of confidentiality arrangements in place international regulatory collaboration; and since 2003, allowing for the exchange of improving governance for the management confidential information as part of their committees and technical working groups. regulatory and scientific processes. However, ►► IGDRP News, 30 August 2017. complete exchange of information was not IPRF News, 30 August 2017 possible under these arrangements. The new confidentiality commitment formally recognises that FDA’s EU counterparts have Medicines labels the authority and demonstrated ability to protect the relevant information. This step Improved labelling in Australia will now allow the sharing of full inspection Australia – The TGA has introduced reports, allowing regulators to make improvements to help align medicine decisions based on findings in each other’s labels with international best practice. inspection reports and to make better use The changes will be implemented over a of their inspection resources to focus on four-year period. Under the new rules, the manufacturing sites of higher risk. names of active ingredients will be updated ►► EMA News, 23 August 2017. to be in line with nonproprietary names used internationally, and medicines names will be displayed more prominently on the IGDRP, IPRF initiatives to join labels. Critical information will be displayed Montreal – The International Generic Drug in a standardized manner and will include Regulators Programme (IGDRP) and the mandatory declaration of some additional International Pharmaceutical Regulators allergens. The changes will also provide for Forum (IPRF) have agreed to consolidate easier dispensing. their collaborative initiatives. The decision ►► TGA. Labelling changes: information for health was taken at the 5th IGDRP meeting, held professionals. 28 July 2017. in Montreal, Canada, on 5–8 June 2017. The joint initiative will be operational in January 2018; the first face-to-face meeting of the

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Under discussion

European Union – Comments are invited European Union – The EMA has launched to an EMA reflection paper on aspects to a public consultation on its proposed consider in development of medicines for recommendations on how best to use older people. Comments are particularly aminoglycoside in animals to invited on the accuracy of tablet breaking, the reduce the risk of resistance.(1) administration of medicines through feeding WHO has classified aminoglycosides as tubes, and on multiple compliance aids and critically important for human health. drug dispensing systems. Comments have also been invited on a draft ►► EMA News, 1 August 2017. reflection paper regarding the off-label use of Closing date: 31 January 2018. veterinary , which contributes to the risk of resistance.(2) ►► (1) EMA News, 25 July 2017. European Union – The EMA has released for Closing date: 31 October 2017 / public consultation a concept paper on the (2) EMA News, 25 July 2017. development and lifecycle of personalized Closing date: 31 January 2018 medicines and companion diagnostics that allow identifying patients who are most likely to benefit from a specific medicine, and those European Union – The EMA has proposed likely to be at increased risk of serious adverse revised guidelines on pharmacovigilance for reactions. Recently revised EU legislation medicines used in children and adolescents foresees cooperation between medicines up to 18 years of age. The revision takes into regulators and EU notified bodies, which account the improved situation with regard conduct the conformity assessment of to off-label use, and considers medicine- diagnostics in the EU. related risks in the context of school and ►► EMA News, 28 July 2017. sports performance, alcohol and nicotine Closing date: 15 November 2017. consumption and possible diversion of medicines to friends. The revised guidance should also be of interest to non-regulatory European Union – A concept on the non- groups such as parents, caregivers and clinical development of radiopharmaceuticals healthcare professionals. has been released for public comment ►► EMA Consultation, 2 August 2017. on the EMA website. The draft guidance Closing date: 13 October 2017. complements existing guidelines, and applies to radiodiagnostics as well as radiotherapeutics. It will focus on targeted European Union – The EMA has released non-clinical programmes for specific a concept paper on revision of its guideline development settings and product types. The on clinical development of . The paper is not intended to duplicate guidance revision of the current guideline is proposed on dosimetry. to incorporate lessons learned in clinical ►► EMA Consultation, 1 August 2017. development of new and improved vaccines Closing date: 31 October 2017. since 2007, as well as aspects of developing

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Under discussion vaccines administered during pregnancy with Canada – Health Canada has opened the main or sole intent of providing a benefit a consultation on proposed changes to to the unborn child. regulations that would make it mandatory ►► EMA Consultation, 23 June 2017. for certain health care institutions to report Closing date: 30 September 2017. serious adverse drug reactions and medical device incidents. ►► Health Canada Consultation, 28 June 2017. Ireland – The Minister for Health of Ireland Closing date: 11 August 2017. has announced the opening of a public consultation on biosimilar medicines. The consultation will inform the development of India – The Office of the Drug Controller Ireland’s first National Biosimilar Medicines General of India has published a notice Policy, with the aim of increasing the use listing some of the steps taken in the past to of these more cost-effective medicines in streamline regulatory procedures, and has Ireland. asked industry to provide feedback on other ►► An Roinn Sláinte / Department of Health. Press proposed approaches that would allow to release, 10 August 2017. streamline the regulatory process further. Consultation closing date: 22 September 2017. ►► CDSCO Notice, 27 June 2017. Closing date: 31 July 2017.

Australia – The TGA is seeking comments on proposed options on whether there is India – The Central Drugs Standard Control a need in Australia for additional naming Organisation of India has published a draft requirements for biological medicines as standard operating procedure for declaring a a way of strengthening traceability and sample as being of substandard quality. pharmacovigilance. ►► CDSCO Notice, 27 June 2017. ►► TGA Consultation, 28 July 2017. Closing date: 31 July 2017. Closing date: 8 September 2017. SOP for handling of Not of Standard Quality (NSQ) drugs samples. Draft.

Geneva – WHO has sought comments on its pilot procedure for WHO prequalification Australia – The TGA has published of similar biotherapeutic products. This feedback on its proposed criteria to identify pilot project is a step towards making some comparable overseas regulators (CORs) as of the most expensive treatments for providers of assessment reports and possible more widely available. The first invitation for work-sharing partners in the assessment of expression of interest to prequalify rituximab- medicine registration applications. A final and trastuzumab-containing products is guideline is expected to be published in planned to be published in October 2017. December 2017. ►► WHO Prequalification News, 21 July 2017. ►► TGA News, 24 July 2017. Closing date: 16 August 2017.

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Approved

L-glutamine for sickle cell disease Class: C1-esterase inhibitor (C1-INH) Product name: Endari® Approval: FDA (orphan drug designation) Dosage form: Oral powder Use: Prevention of hereditary angioedema attacks Class: Amino acid in adolescent and adult patients Approval: FDA (orphan drug designation) Benefits: Reduced number of attacks, compared Use: To reduce the acute complications of sickle to placebo. cell disease in adults and children 5 years of age Notes: Hereditary angioedema causes attacks of and older rapid swelling of the hands, feet, limbs, face, Benefits: Fewer and shorter hospital visits for intestinal tract or airway. These attacks can sickle cell crises, fewer occurrences of acute occur spontaneously, or can be triggered by chest syndrome than with placebo. stress, surgery or infection. Notes: Only one other medicine is approved in the The product is not suitable to treat acute U.S. for sickle cell disease. attacks. ►► FDA News release, 7 July 2017. ►► FDA News release, 22 June 2017.

Betrixaban to prevent venous Meropenem and vaborbactam for thromboembolism in certain patients complicated urinary tract infection Product name: BevyxXa® Product name: Vabomere® Dosage form: Capsules Dosage form: Sterile powder for injection for Class: , Factor Xa inhibitor intravenous use Approval: FDA Class: Combination of a penem antibacterial Use: Prophylaxis of venous thromboembolism (meropenem) and a beta-lactamase inhibitor (VTE) in adult patients hospitalized for (vaborbactam) an acute medical illness who are at risk for Approval: FDA (priority review, qualified thromboembolic complications infectious disease product (QIDP) designation) Benefits: In a clinical trial, betrixaban was more Use: Treatment of adults with complicated urinary efficacious than enoxaparin in preventing tract infections (cUTI). To reduce the risk of thrombotic events. drug-resistance, the product should be used Safety information: Patients treated with only to treat or prevent infections that are betrixaban who are receiving neuraxial proven or strongly suspected to be caused by anesthaesia or undergoing spinal puncture are susceptible bacteria. at risk of spinal/epidural haematoma. The risk Benefits: Additional treatment option for cUTI may be increased by the use of in-dwelling Safety information: This product can cause allergic epidural catheters or the concomitant use reactions and seizures. It should not be used of medical products affecting haemostasis. in patients with a history of anaphylaxis in These haematomas may result in long-term or response to beta-lactams permanent paralysis. ►► FDA Press release, 29 August 2017. ►► Prescribing information for BevyxXa®, revised 6/2017. Delafloxacin for acute bacterial skin infections C1 esterase inhibitor (human) to prevent Product name: Baxdela® hereditary angioedema Dosage form: Tablets; injection for intravenous use Product name: Haegarda® Class: Fluoroquinolone Dosage form: Lyophilized concentrate for Approval: FDA subcutaneous injection

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Approved

Use: Treatment of acute bacterial skin and skin Approval: EMA (accelerated approval); structure infections caused by designated FDA (priority review, breakthrough therapy) susceptible bacteria. Delafloxacin should be Use: Treatment of chronic hepatitis C virus used only to treat infections that are proven or (HCV) infection in adults strongly suspected to be caused by bacteria. Benefits: Highly effective against all genotypes of Benefits: No less effective than a combination of HCV. Can be used in patients in whom prior vancomycin and aztreonam in two multi- treatment with other direct-acting antivirals centre clinical trials. has failed. Safety information: Like other fluoroquinolones Safety information: The product is contra­indicated delafloxacin is associated with: (1) a risk of in patients treated with rifampicin. disabling and potentially irreversible serious ►► EMA Press release, 23 June 2017. adverse reactions that can occurred together, FDA News release, 18 July 2017. including tendinitis and tendon rupture, peripheral neuropathy and central nervous system effects; and (2) a risk of exacerbation of for relapsing multiple sclerosis myasthenia gravis. Product name: Mavenclad ® ►► Prescribing information for Baxdela®, revised Dosage form: Tablets 6/2017. Class: Antimetabolite; ATC code: L01BB04 Approval: EMA Use: Treatment of adult patients with highly Glecaprevir and pibrentasvir for active relapsing multiple sclerosis as defined by hepatitis C clinical or imaging features. Product name: EU: Maviret®; U.S.: Mavyret® Benefits: Ability to reduce the frequency of Dosage form: Film-coated tablets relapses and to delay disease progression Class: Fixed-dose combination of two direct- Safety information: Cladribine can cause acting antivirals, a HCV NS3/4A protease lymphopenia, which can be severe and long- inhibitor (glecaprevir), and HCV NS5A lasting, and infections including herpes zoster. inhibitor (pibrentasvir) ►► EMA/CHMP Summary of opinion, 22 June 2017. Approval: EMA (accelerated approval); FDA (priority review, breakthrough therapy) Use: Treatment of chronic hepatitis C virus and for two (HCV) infection in adults types of acute myeloid leukaemia (AML) Benefits: Highly effective against all genotypes of Product name: Vyxeos® HCV. Can be used in patients with severe renal Dosage form: Liposome for injection for impairment, including in those on dialysis. intravenous use ►► EMA Press release, 23 June 2017. Class: Fixed-dose combination of an FDA News release, 3 August 2017. (daunorubicin) and a metabolic inhibitor (cytarabine) Sofosbuvir, velpatasvir and voxilaprevir Approval: FDA (priority review, breakthrough for hepatitis C therapy; orphan drug designation) Product name: Vosevi® Use: Treatment of adult patients with newly Dosage form: Film-coated tablets diagnosed therapy-related AML, or AML with Class: Fixed-dose combination of three direct myelodysplasia-related changes antivirals: a analogue non- Benefits: Longer overall survival than with structural protein NS5B polymerase inhibitor separate treatments of daunorubicin and (sofosbuvir), (an HCV NS5A inhibitor cytarabine. (velpatasvir) and a novel pangenotypic HCV Safety information: The product has been NS3/4A protease inhibitor (voxilaprevir). associated with serious or fatal bleeding events. This product should not be interchanged

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Approved

with other daunorubicin- and/or cytarabine- Use: Treatment of adult patients with relapsed or containing products. refractory acute myeloid leukaemia (AML) Note: This is the first FDA-approved treatment who have a specific genetic mutation specifically for patients with either of these two Benefits: Ability to achieve complete remission in types of high-risk AML. some patients and a reduction in the need for ►► FDA News release, 3 August 2017. both red cell and platelet transfusions lasting several months. Safety information: An adverse reaction known as Gemtuzumab ozogamicin for acute differentiation syndrome can occur and can be myeloid leukaemia (AML) fatal if not treated. If differentiation syndrome Product name: Mylotarg® is suspected, patients should be treated with Dosage form: Lyophilized cake or powder for and closely monitored until injection symptoms resolve. Class: CD33-directed antibody-drug conjugate Note: The product is approved for use with Approval: FDA (orphan drug designation) a companion diagnostic, the RealTime Use: Treatment of adults with newly diagnosed IDH2 Assay, which is used to detect specific CD33-positive AML; treatment of patients mutations in patients’ IDH2 gene. aged 2 years and older with relapsed or ►► FDA News release, 1 August 2017. refractory CD33-positive AML. Benefits: Longer event-free survival period than with alone; longer median Neratinib to reduce risk of breast cancer overall survival than with best supportive care. Product name: Nerlynx® Safety information: Severe side effects include Dosage form: Tablets liver damage, hepatic veno-occlusive disease, Class: Kinase inhibitor low blood counts, infections, infusion-related Approval: FDA reactions and severe bleeding. Use: For the extended adjuvant treatment of early- Notes: (1) Mylotarg® was originally received stage, HER2-positive breast cancer in adult accelerated FDA-approval in May 2000 as a patients previously treated with trastuzumab stand-alone treatment for older patients with Benefits: Reduced risk of breast cancer recurrence CD33-positive AML who had experienced Safety information: Severe potential adverse effects a relapse. The product was voluntarily include diarrhoea and liver damage. Patients withdrawn from the market after subsequent should be given loperamide for the first 56 confirmatory trials failed to verify clinical days of treatment and as needed thereafter, benefit and demonstrated safety concerns, together with additional antidiarrheals, fluids including a high number of early deaths. The and electrolytes as clinically indicated to help 2017 approval includes a lower recommended manage diarrhoea. Neratinib may cause harm dose, a different schedule in combination with to a developing foetus or a newborn child. chemotherapy or on its own, and a new patient ►► FDA News release, 17 July 2017. population. ►► FDA News release, 1 September 2017. Tivozanib for kidney cancer Product name: Fotivda® Enasidenib for certain types of AML Dosage form: Hard capsules Product name: Idhifa® Class: ; Dosage form: Tablets ATC code: L01XE34 Class: Isocitrate dehydrogenase-2 inhibitor Approval: EMA Approval: FDA (priority review; orphan drug Use: First line-treatment of adult patients with designation) advanced renal cell carcinoma (RCC) and treatment of certain adult patients following

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Approved

disease progression after one prior cytokine paediatric eye drops for rare therapy for advanced RCC. eye allergy in children Benefits: Ability to improve progression-free Product name: Verkazia® survival in patients with advanced disease Dosage form: Eye drops ►► EMA/CHMP Summary of opinion, 22 June 2017. Class: Immunosuppressant; ATC code: S01XA18 Approval: EMA (orphan designation; accelerated assessment) Guselkumab for plaque psoriasis Use: Treatment of severe vernal kerato­ Product name: Tremfya® conjunctivitis in children from 4 years of age Dosage form: Subcutaneous injection and adolescents Class: Interleukin-23 blocker; Benefits: Ability to improve ocular surface ATC code (temporary): L04AC16 damage and reduce symptoms of severe Approval: FDA vernal keratoconjunctivitis in children and Use: Treatment of adult patients with moderate to adolescent patients. severe plaque psoriasis who are candidates for ►► EMA Press release, 21 July 2017. systemic therapy or phototherapy. Benefits: Additional treatment option for psoriasis. Lutetium oxodotreotide for certain Safety information: Guselkumab may increase the gastroenteric  risk of infection. Patients should be evaluated Product name: Lutathera® for tuberculosis before starting treatment Dosage form: Solution for infusion ►► FDA prescribing information for Tremfya®; Class: Radiolabelled peptide targeting revised July 2017. subtype 2 somatostatin (sst2) receptors; ATC code: V10XX04 Approval: EMA (orphan designation) Benznidazole for Chagas disease Use: Treatment of gastro-entero-pancreatic Dosage form: Oral tablets neuroendocrine tumours Class: Nitroimidazole derivative; Benefits: Ability to improve progression-free ATC code: P01CA02 survival compared with octreotide long-acting Approval: FDA (accelerated approval, priority release (LAR), a somatostatin receptor agonist, review; orphan product designation) in patients with certain types of tumours. Use: Treatment of children aged 2 to 12 years with ►► EMA/CHMP Summary of opinion, 20 July 2017. Chagas disease Benefits: Significantly more seroconversions from positive to negative antibody test compared Gene cell therapy with placebo. Safety information: Benznidazole can cause Tisagenlecleucel for certain leukaemias serious skin reactions, nervous system effects Product name: Kymriah® and bone marrow depression. Based on Dosage form: Autologous CAR T cells for infusion findings from animal studies, benznidazole Class: Genetically-modified autologous T-cell could cause foetal harm. immunotherapy. chimeric antigen receptor Notes: Chagas disease is a parasitic infection that (CAR) designed to kill B-cells with a C19 can cause serious heart illness and can affect surface antigen. swallowing and digestion in the long term. Approval: FDA (fast track, priority review and This is the first FDA-approved treatment for breakthrough therapy designations) Chagas disease. Use: Treatment of children and young adults ►► FDA Press release, 29 August 2017. up to 25 years of age with B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in second or later relapse..

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Approved

Benefits: Remission rate of 83% within three (2) months in a clinical trial. Product name: Cyltezo® Safety information: The product carries a Reference product: Humira® boxed warning about the risk of cytokine Approval: FDA release syndrome (CRS), which causes high Use: Treatment of rheumatoid arthritis, juvenile fever and flu-like symptoms, and the risk idiopathic arthritis, psoriatic arthritis, of neurological events. Both these events ankylosing spondylitis, adult Crohn’s disease can be life-threatening. Other severe side ulcerative colitis and plaque psoriasis. effects of Kymriah include serious infections, ►► Prescribing information for Cyltezo®. Revised hypotension, acute kidney injury, fever, and August 2017. hypoxia. Most symptoms appear within one to 22 days following infusion Note: This is the first gene therapy available in the Early access United States. ►► FDA Press release, 30 August 2017. Idebenone for Duchenne’s muscular dystrophy Product name: Raxone® Biosimilars Dosage form: Tablets Class: Antioxydant Bevacizumab Approval: MHRA Early Access to Medicines Product name: Mvasi® (bevacizumab-awwb) Scheme (EAMS) Reference product: Avastin® Use: Treatment of patients aged 10 years and Approval: FDA older with Duchenne’s muscular dystrophy not Use: Treatment of adult patients with certain currently taking and showing colorectal, lung, brain, kidney and cervical clear signs of deteriorating lung function. cancers. Benefits: Ability to slow the decline of respiratory Safety information: Like Avastin®, Mvasi® caries function a Boxed Warning about an increased risk of Note: The product is licensed in the U.K. for gastrointestinal perforations; surgery and the treatment of Leber’s hereditary optic wound healing complications; and severe neuropathy, a rare eye condition. or fatal pulmonary, gastrointestinal, central ►► MHRA Public assessment report. nervous system and vaginal bleeding. Note: Bevacizumab-awwb is the first biosimilar approved in the U.S. for the treatment of Extensions of indications cancer. ►► FDA News release, 14 September 2017. Ibrutinib for graft-versus-host disease Product name: Imbruvica® Approval: FDA (priority review, breakthrough therapy; orphan drug designation) (1) Newly approved use: Treatment of chronic graft- Product name: Imraldi® versus-host disease (cGVHD) Reference product: Humira® Note: cGVHD is a serious and life-threatening Approval: EMA condition that may occur in patients Use: Treatment of rheumatoid arthritis, juvenile with blood cancer who receive a stem cell idiopathic arthritis, axial spondyloarthritis, transplant. This is the first FDA-approved psoriatic arthritis, psoriasis, paediatric plaque therapy for cGVHD. psoriasis, hidradenitis suppurativa, Crohn’s ►► FDA News release, 2 August 2017. å disease, paediatric Crohn’s disease, ulcerative colitis and uveitis. ►► EMA/CHMP Summary of opinion, 22 June 2017.

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