DOCSLIB.ORG

2020 End of the Year Summary Report-2 FDA Approved 13 New Large Molecule Drugs and 2 New Gene Therapy Products

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
2020 End of the Year Summary Report-2 FDA Approved 13 New Large Molecule Drugs and 2 New Gene Therapy Products 2020 End of the Year Summary Report-2 FDA Approved 13 New Large Molecule Drugs and 2 New Gene Therapy Products BLA Regulatory, LLC (Gaithersburg, MD) provides the following summary of 2020 FDA approved new large molecule (LM) drugs and gene therapy products. Reviewers/Readers are suggested to open FDA links for detailed information and accuracy. Number of approved LM drugs and Gene Therapies in sub-categories Therapeutic Area Formulation Regulatory Program Oncology 6 IV Solution 11 Breakthrough Therapy 2 Genetic disorder 3 Subcutaneous Solution 3 Fast Track 1 Neurology 2 Lyophilized powder 1 Orphan Drug 11 Infection 2 Priority Review 3 Metabolic disease 1 Ophthalmology 1 Page 1 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 1 TEPEZZA Horizon Treatment of IGF-1R Priority "There were two randomized, double-masked, placebo-controlled trials that FDA link 1/21 (teprotumum Pharma Thyroid Eye Review, provided data for approval of TEPEZZA. ab-trbw) Ireland, Disease Fast Track, Ltd. Breakthrou Patients with active thyroid eye disease received TEPEZZA by intravenous Lyophilized gh Therapy infusions (10 mg/kg for first infusion and 20 mg/kg for the remaining 7 powder for Designation infusions) or placebo infusions every 3 weeks for a total of 8 infusions. intravenous and Orphan infusion Drug The main outcome measure was the proptosis responder rate at week 24 designation defined as the percentage of patients with ≥2 mm reduction in proptosis in LM the study eye from baseline, without deterioration in the non-study eye (≥2 mm increase)." 2 VYEPTI Lundbeck The preventive CGRP N/A "The efficacy and safety of VYEPTI were evaluated in two similar, FDA Link 2/21 (eptinezuma Seattle treatment of randomized, multicenter, placebo-controlled clinical trials. b-jjmr) BioPharm migraine in adults aceuticals, Trial 1 enrolled patients with episodic migraine. VYEPTI or placebo was intravenous Inc. administered by intravenous infusion every 3 months for 12 months; infusion however, the primary endpoint was measured at 12 weeks. LM Trial 2 enrolled patients with chronic migraine. VYEPTI or placebo was administered by intravenous infusion every 3 months for 6 months; the primary endpoint was measured at 12 weeks. The primary efficacy endpoint for both trials was the change from baseline in mean monthly migraine days over Months 1-3. Secondary endpoints included the percentages of patients with 50% or greater and 75% or greater reductions from baseline in monthly migraine days over Months 1-3." 3 SARCLISA® Sanofi The treatment of CD38 Orphan The efficacy and safety of SARCLISA in combination with pomalidomide and FDA Link 3/2 (isatuximab- Aventis adult patients with Drug design low-dose dexamethasone were evaluated in a multicenter, randomized, irfc) US LLC multiple myeloma ation open-label trial in patients who had had received at least two prior therapies who have including lenalidomide and a proteasome inhibitor. Patients were randomized intravenous received at least in a 1:1 ratio to receive either SARCLISA in combination with pomalidomide infusion two prior and low-dose dexamethasone or pomalidomide and low-dose therapies dexamethasoneTreatment was administered in both groups in 28-day cycles LM including until disease progression or unacceptable toxicity. The primary endpoint was lenalidomide and progression-free survival (PFS). PFS results were assessed by an a proteasome Independent Response Committee based on central laboratory data for M- inhibitor protein and central radiologic imaging review using the International Myeloma Working Group (IMWG) criteria. Page 2 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 4 TRODELVY Immunom The treatment of TACSTD N/A "There was one multicenter, single-arm, trial that enrolled patients with FDA Link 4/22 ™ edics, Inc adult patients with 2 metastatic triple-negative breast cancer (mTNBC) who had received at least (sacituzumab metastatic triple- two prior treatments for metastatic disease. govitecan- negative breast hziy) cancer (mTNBC) Patients received TRODELVY 10 mg/kg intravenously on Days 1 and 8 of a who have 21-day treatment cycle. Tumor imaging was obtained every 8 weeks, and Intravenous received at least treatment continued until disease progression or intolerance to the therapy. injection two prior therapies for Major efficacy outcome measures were investigator assessed ORR using LM metastatic RECIST 1.1 and duration of response." disease. 5 UPLIZNA™ Viela Bio, The treatment of B- Orphan There was one trial of 197 days duration that evaluated the benefits and side FDA Link 6/11 (inebilizumab Inc. neuromyelitis lymphocy Drug effects of UPLIZNA. The trial enrolled adult patients with NMOSD who were -cdon) optica spectrum te designation positive for anti-aquaporin-4 antibody. Patients received at random either disorder antigen UPLIZNA or placebo infusions according to the schedule. Neither the Intravenous (NMOSD) in adult CD19 patients nor the healthcare providers knew which treatment was being given. injection patients who are anti-aquaporin-4 LM (AQP4) antibody positive 6 MONJUVI® MorphoSy The treatment of B- N/A; "The safety and efficacy of MONJUVI were established in an open label, FDA Link 7/31 (tafasitamab- s US Inc. adult patients with lymphocy Orphan multicenter trial in adult patients with relapsed or refractory DLBCL after 1 to cxix) relapsed or te 3 prior systemic therapies. Enrolled patients were not candidates for high refractory diffuse antigen dose chemotherapy followed by autologous stem cell transplantation. Intravenous large B-cell CD19 lymphoma Patients received MONJUVI 12 mg/kg intravenously in combination with LM (DLBCL) not lenalidomide (25 mg orally on Days 1 to 21 of each 28-day cycle) for otherwise maximum of 12 cycles, followed by MONJUVI as a monotherapy until specified, disease progression or unacceptable toxicity. including DLBCL arising from low Efficacy was established based on best overall response rate, defined as the grade lymphoma, proportion of complete and partial responders, and duration of response as and who are not determined by an IRC using the International Working Group Response eligible for Criteria." autologous stem cell transplant (ASCT) Page 3 of 6 If re-sharing and re-printing, please indicate BLA as the source of this summary. Number New Drug Sponsor indication Drug Regulatory Unique clinical design Details Approve formulation target special Date program 7 BLENREP GlaxoSmit The treatment of TNFRSF N/A; "The efficacy and safety of BLENREP were evaluated in a multicenter, open- FDA Link 8/5 (belantamab hKline adult patients with 17 Orphan label trial of patients with relapsed refractory multiple myeloma who had mafodotin- Intellectual relapsed or Drug previously received 3 or more anti-myeloma treatment regimens, and whose blmf) Property refractory multiple designation disease no longer responded to an immunomodulatory agent and a Developm myeloma who proteasome inhibitor. Treatment was administered as an intravenous Intravenous ent Ltd. have received at infusion once every 3 weeks until disease progression or unacceptable injection least 4 prior toxicity. therapies LM including an anti- The primary endpoint was ORR, defined as the proportion of patients with a CD38 monoclonal PR or better, as assessed by an IRC based on the IMWG Uniform Response antibody, a Criteria for Multiple Myeloma." proteasome inhibitor, and an immunomodulator y agent. 8 VILTEPSO Nippon The treatment of DMD Priority "VILTEPSO for the treatment of DMD was evaluated in two clinical trials. FDA Link 8/12 (viltolarsen) Shinyaku Duchenne gene Review, Enrolled patients had a genotypically confirmed DMD diagnosis (a confirmed Co., Ltd. muscular (exon 53 Orphan mutation of the DMD gene that is amenable to exon 53 skipping) and were Intravenous dystrophy (DMD) viltolarse Drug on a stable dose of corticosteroids for at least 3 months prior to enrollment. injection in patients who n target designation have a confirmed site) Trial 1 was a multicenter, 2 period, dose-finding trial. During the initial, Gene mutation of the double blind period (first 4 weeks), which evaluated the acute safety of Therapy DMD gene that is VILTEPSO, patients were randomized to VILTEPSO or placebo. All patients amenable to exon were then re-randomized and received 20 weeks of open-label VILTEPSO 53 skipping. 40 mg/kg once weekly (0.5 times the recommended dosage) or 80 mg/kg once weekly. The primary efficacy endpoint was the change from baseline in the dystrophin protein level (measured as % of normal) at Week 25. Trial 2 was a multicenter, parallel-group, open-label, dose-finding trial. Patients were assigned to receive intravenous VILTEPSO 40 mg/kg once weekly (0.5 times the recommended dose) (N=8) or 80 mg/kg once weekly (N=8) for 24 weeks." 9 ENSPRYNG Genentec The treatment of Interleuki N/A; "The safety and efficacy of ENSPRYNG were established in two FDA Link 8/14 ™ h, Inc. neuromyelitis n-6 Orphan randomized, placebo- controlled trials of patients with NMOSD. (satralizuma optica spectrum receptor Drug b-mwge) disorder subunit designation In both trials, patients were randomized to and received ENSPRYNG or (NMOSD) in adult alpha placebo. In Trial 2, all patients were also
Load more

Recommended publications
  • DRUGS REQUIRING PRIOR AUTHORIZATION in the MEDICAL BENEFIT Page 1
    Effective Date: 08/01/2021 DRUGS REQUIRING PRIOR AUTHORIZATION IN THE MEDICAL BENEFIT Page 1 Therapeutic Category Drug Class Trade Name Generic Name HCPCS Procedure Code HCPCS Procedure Code Description Anti-infectives Antiretrovirals, HIV CABENUVA cabotegravir-rilpivirine C9077 Injection, cabotegravir and rilpivirine, 2mg/3mg Antithrombotic Agents von Willebrand Factor-Directed Antibody CABLIVI caplacizumab-yhdp C9047 Injection, caplacizumab-yhdp, 1 mg Cardiology Antilipemic EVKEEZA evinacumab-dgnb C9079 Injection, evinacumab-dgnb, 5 mg Cardiology Hemostatic Agent BERINERT c1 esterase J0597 Injection, C1 esterase inhibitor (human), Berinert, 10 units Cardiology Hemostatic Agent CINRYZE c1 esterase J0598 Injection, C1 esterase inhibitor (human), Cinryze, 10 units Cardiology Hemostatic Agent FIRAZYR icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent HAEGARDA c1 esterase J0599 Injection, C1 esterase inhibitor (human), (Haegarda), 10 units Cardiology Hemostatic Agent ICATIBANT (generic) icatibant J1744 Injection, icatibant, 1 mg Cardiology Hemostatic Agent KALBITOR ecallantide J1290 Injection, ecallantide, 1 mg Cardiology Hemostatic Agent RUCONEST c1 esterase J0596 Injection, C1 esterase inhibitor (recombinant), Ruconest, 10 units Injection, lanadelumab-flyo, 1 mg (code may be used for Medicare when drug administered under Cardiology Hemostatic Agent TAKHZYRO lanadelumab-flyo J0593 direct supervision of a physician, not for use when drug is self-administered) Cardiology Pulmonary Arterial Hypertension EPOPROSTENOL (generic)
    [Show full text]
  • Oregon Medicaid Pharmaceutical Services Prior Authorization Criteria
    Oregon Medicaid Pharmaceutical Services Prior Authorization Criteria HEALTH SYSTEMS DIVISION Prior authorization (PA) criteria for fee-for-service prescriptions for Oregon Health Plan clients March 1, 2021 Contents Contents ................................................................................................................................................................ 2 Introduction........................................................................................................................................................... 7 About this guide ......................................................................................................................................... 7 How to use this guide ................................................................................................................................. 7 Administrative rules and supplemental information .................................................................................. 7 Update information............................................................................................................................................... 8 Effective March 1, 2021 ............................................................................................................................ 8 Substantive updates and new criteria ............................................................................................. 8 Clerical changes ............................................................................................................................
    [Show full text]
  • Refreshing the Biologic Pipeline 2020
    news feature Credit: Science Lab / Alamy Stock Photo Refreshing the biologic pipeline 2020 In the absence of face-to-face meetings, FDA and industry implemented regulatory workarounds to maintain drug and biologics approvals. These could be here to stay. John Hodgson OVID-19 might have been expected since 1996) — a small miracle in itself “COVID-19 confronted us with the need to severely impair drug approvals (Fig. 1 and Table 1). to better triage sponsors’ questions,” says Cin 2020. In the event, however, To the usual crop of rare disease and Peter Marks, the director of the Center for industry and regulators delivered a small genetic-niche cancer treatments, 2020 Biologics Evaluation and Research (CBER) miracle. They found workarounds and also added a chimeric antigen receptor at the FDA. “That was perhaps the single surrogate methods of engagement. Starting (CAR)-T cell therapy with a cleaner biggest takeaway from the pandemic related in January 2020, when the outbreak veered manufacturing process and the first to product applications.” Marks says that it westward, the number of face-to face approved blockbuster indication for a became very apparent with some COVID- meetings declined rapidly; by March, small-interfering RNA (siRNA) — the 19-related files that resolving a single they were replaced by Webex and Teams. European Medicines Agency’s (EMA) issue can help a sponsor enormously and (Secure Zoom meeting are to be added registration of the RNA interference accelerate the development cycle. Before this year.) And remarkably, by 31 December, (RNAi) therapy Leqvio (inclisiran) for COVID-19, it was conceivable that a small the US Food and Drug Administration cardiovascular disease.
    [Show full text]
  • CDER Breakthrough Therapy Designation Approvals Data As of December 31, 2020 Total of 190 Approvals
    CDER Breakthrough Therapy Designation Approvals Data as of December 31, 2020 Total of 190 Approvals Submission Application Type and Proprietary Approval Use Number Number Name Established Name Applicant Date Treatment of patients with previously BLA 125486 ORIGINAL-1 GAZYVA OBINUTUZUMAB GENENTECH INC 01-Nov-2013 untreated chronic lymphocytic leukemia in combination with chlorambucil Treatment of patients with mantle cell NDA 205552 ORIGINAL-1 IMBRUVICA IBRUTINIB PHARMACYCLICS LLC 13-Nov-2013 lymphoma (MCL) Treatment of chronic hepatitis C NDA 204671 ORIGINAL-1 SOVALDI SOFOSBUVIR GILEAD SCIENCES INC 06-Dec-2013 infection Treatment of cystic fibrosis patients age VERTEX PHARMACEUTICALS NDA 203188 SUPPLEMENT-4 KALYDECO IVACAFTOR 21-Feb-2014 6 years and older who have mutations INC in the CFTR gene Treatment of previously untreated NOVARTIS patients with chronic lymphocytic BLA 125326 SUPPLEMENT-60 ARZERRA OFATUMUMAB PHARMACEUTICALS 17-Apr-2014 leukemia (CLL) for whom fludarabine- CORPORATION based therapy is considered inappropriate Treatment of patients with anaplastic NOVARTIS lymphoma kinase (ALK)-positive NDA 205755 ORIGINAL-1 ZYKADIA CERITINIB 29-Apr-2014 PHARMACEUTICALS CORP metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib Treatment of relapsed chronic lymphocytic leukemia (CLL), in combination with rituximab, in patients NDA 206545 ORIGINAL-1 ZYDELIG IDELALISIB GILEAD SCIENCES INC 23-Jul-2014 for whom rituximab alone would be considered appropriate therapy due to other co-morbidities
    [Show full text]
  • Antibodies to Watch in 2021 Hélène Kaplona and Janice M
    MABS 2021, VOL. 13, NO. 1, e1860476 (34 pages) https://doi.org/10.1080/19420862.2020.1860476 PERSPECTIVE Antibodies to watch in 2021 Hélène Kaplona and Janice M. Reichert b aInstitut De Recherches Internationales Servier, Translational Medicine Department, Suresnes, France; bThe Antibody Society, Inc., Framingham, MA, USA ABSTRACT ARTICLE HISTORY In this 12th annual installment of the Antibodies to Watch article series, we discuss key events in antibody Received 1 December 2020 therapeutics development that occurred in 2020 and forecast events that might occur in 2021. The Accepted 1 December 2020 coronavirus disease 2019 (COVID-19) pandemic posed an array of challenges and opportunities to the KEYWORDS healthcare system in 2020, and it will continue to do so in 2021. Remarkably, by late November 2020, two Antibody therapeutics; anti-SARS-CoV antibody products, bamlanivimab and the casirivimab and imdevimab cocktail, were cancer; COVID-19; Food and authorized for emergency use by the US Food and Drug Administration (FDA) and the repurposed Drug Administration; antibodies levilimab and itolizumab had been registered for emergency use as treatments for COVID-19 European Medicines Agency; in Russia and India, respectively. Despite the pandemic, 10 antibody therapeutics had been granted the immune-mediated disorders; first approval in the US or EU in 2020, as of November, and 2 more (tanezumab and margetuximab) may Sars-CoV-2 be granted approvals in December 2020.* In addition, prolgolimab and olokizumab had been granted first approvals in Russia and cetuximab saratolacan sodium was first approved in Japan. The number of approvals in 2021 may set a record, as marketing applications for 16 investigational antibody therapeutics are already undergoing regulatory review by either the FDA or the European Medicines Agency.
    [Show full text]
  • Chemotherapy: Drugs E-O Policy (Chemo Drug E-O)
    chemo drug e-o 1 Chemotherapy: Drugs E-O Policy Page updated: September 2020 This section contains policy related to billing for injection services, listed in alphabetical order by generic drug name or drug type. For general billing policy information regarding injections services, refer to the Chemotherapy: An Overview section in this manual. Additional policy information for chemotherapy drug services can be found in the Chemotherapy: Drugs A-D Policy and Chemotherapy: Drugs P-Z Policy sections in this manual. Elotuzumab Elotuzumab is a humanized IgG1 monoclonal antibody that specifically targets the SLAMF7 (signaling lymphocytic activation molecule family member 7) protein. SLAMF7 is expressed on myeloma cells independent of cytogenetic abnormalities. SLAMF7 is also expressed on natural killer cells, plasma cells and at lower levels on specific immune cell subsets of differentiated cells within the hematopoietic lineage. Elotuzumab directly activates natural killer cells through both the SLAMF7 pathway and Fc receptors. Elotuzumab also targets SLAMF7 on myeloma cells and facilitates the interaction with natural killer cells to mediate the killing of myeloma cells through antibody-dependent cellular cytotoxicity (ADCC). Indications Elotuzumab is indicated in combination with lenalidomide and dexamethasone for the treatment of patients ages 18 years or older, with multiple myeloma who have received one to three prior therapies. Pre-medicate with dexamethasone, diphenhydramine, ranitidine and acetaminophen. Advise patients that lenalidomide has the potential to cause fetal harm. Authorization An approved Treatment Authorization Request (TAR) is required for reimbursement. The TAR must state that the treatment is for a patient with multiple myeloma who has received one to three prior therapies.
    [Show full text]
  • Antibodies for the Treatment of Brain Metastases, a Dream Or a Reality?
    pharmaceutics Review Antibodies for the Treatment of Brain Metastases, a Dream or a Reality? Marco Cavaco, Diana Gaspar, Miguel ARB Castanho * and Vera Neves * Instituto de Medicina Molecular, Faculdade de Medicina, Universidade de Lisboa, Av. Prof. Egas Moniz, 1649-028 Lisboa, Portugal * Correspondence: [email protected] (M.A.R.B.C.); [email protected] (V.N.) Received: 19 November 2019; Accepted: 28 December 2019; Published: 13 January 2020 Abstract: The incidence of brain metastases (BM) in cancer patients is increasing. After diagnosis, overall survival (OS) is poor, elicited by the lack of an effective treatment. Monoclonal antibody (mAb)-based therapy has achieved remarkable success in treating both hematologic and non-central-nervous system (CNS) tumors due to their inherent targeting specificity. However, the use of mAbs in the treatment of CNS tumors is restricted by the blood–brain barrier (BBB) that hinders the delivery of either small-molecules drugs (sMDs) or therapeutic proteins (TPs). To overcome this limitation, active research is focused on the development of strategies to deliver TPs and increase their concentration in the brain. Yet, their molecular weight and hydrophilic nature turn this task into a challenge. The use of BBB peptide shuttles is an elegant strategy. They explore either receptor-mediated transcytosis (RMT) or adsorptive-mediated transcytosis (AMT) to cross the BBB. The latter is preferable since it avoids enzymatic degradation, receptor saturation, and competition with natural receptor substrates, which reduces adverse events. Therefore, the combination of mAbs properties (e.g., selectivity and long half-life) with BBB peptide shuttles (e.g., BBB translocation and delivery into the brain) turns the therapeutic conjugate in a valid approach to safely overcome the BBB and efficiently eliminate metastatic brain cells.
    [Show full text]
  • Cantargia Strengthens the Company Management Team
    Press release Cantargia AB 556791-6019 1 September 2021 Cantargia strengthens the company management team Cantargia AB (publ) (Cantargia) announced today that Nedjad Losic has been employed as VP Biometrics at Cantargia from 1 September 2021. He will be part of the management team where he will contribute with his extensive experience, from e.g. development of daratumumab at Genmab, of antibodies for cancer treatment. Cantargia's research portfolio has developed well, and preparations are underway to advance to late stage clinical development phase. In connection with this, the management team has been strengthened in statistics, and data management, two future key activities. Nedjad Losic has unique competence in these areas and will take responsibility for both. Nedjad Losic, who most recently held a position at Y-mAbs Therapeutics Inc., has 25 years of experience in pharmaceutical drug development, including 16 years with antibodies in oncology. He had key roles in the development and market approvals of daratumumab (multiple myeloma), ofatumumab (chronic lymphocytic leukemia) and naxitamab (neuroblastoma). Nedjad has also been CEO at Spadille Sweden as well as Board Member of Genmab A/S. He holds a M. Sc. in Mathematics, from the University of Lund. “We are pleased to have Nedjad in Cantargia's organization. With his extensive experience from development of antibodies for cancer treatment and interactions with regulatory authorities, he will have an important function in the company when we take the next step in the clinical development program with our main project nadunolimab," says Göran Forsberg, CEO of Cantargia. For further information, please contact Göran Forsberg, CEO Telephone: +46 (0)46-275 62 60 E-mail: [email protected] The information was submitted for publication at 09.00 CET on 1 September 2021.
    [Show full text]
  • Antibodies to Watch in 2021 Hélène Kaplona and Janice M
    MABS 2021, VOL. 13, NO. 1, e1860476 (34 pages) https://doi.org/10.1080/19420862.2020.1860476 PERSPECTIVE Antibodies to watch in 2021 Hélène Kaplona and Janice M. Reichert b aInstitut De Recherches Internationales Servier, Translational Medicine Department, Suresnes, France; bThe Antibody Society, Inc., Framingham, MA, USA ABSTRACT ARTICLE HISTORY In this 12th annual installment of the Antibodies to Watch article series, we discuss key events in antibody Received 1 December 2020 therapeutics development that occurred in 2020 and forecast events that might occur in 2021. The Accepted 1 December 2020 coronavirus disease 2019 (COVID-19) pandemic posed an array of challenges and opportunities to the KEYWORDS healthcare system in 2020, and it will continue to do so in 2021. Remarkably, by late November 2020, two Antibody therapeutics; anti-SARS-CoV antibody products, bamlanivimab and the casirivimab and imdevimab cocktail, were cancer; COVID-19; Food and authorized for emergency use by the US Food and Drug Administration (FDA) and the repurposed Drug Administration; antibodies levilimab and itolizumab had been registered for emergency use as treatments for COVID-19 European Medicines Agency; in Russia and India, respectively. Despite the pandemic, 10 antibody therapeutics had been granted the immune-mediated disorders; first approval in the US or EU in 2020, as of November, and 2 more (tanezumab and margetuximab) may Sars-CoV-2 be granted approvals in December 2020.* In addition, prolgolimab and olokizumab had been granted first approvals in Russia and cetuximab saratolacan sodium was first approved in Japan. The number of approvals in 2021 may set a record, as marketing applications for 16 investigational antibody therapeutics are already undergoing regulatory review by either the FDA or the European Medicines Agency.
    [Show full text]
  • (CMS) Healthcare Common Procedure Coding System (HCPCS) Application Summaries and Coding Decisions First Quarter 2021 Coding Cycle for Drug and Biological Products
    Centers for Medicare & Medicaid Services (CMS) Healthcare Common Procedure Coding System (HCPCS) Application Summaries and Coding Decisions First Quarter 2021 Coding Cycle for Drug and Biological Products This document presents, in request number sequence, a summary of each HCPCS code application and CMS’ coding decision for each application processed in CMS’ First Quarter 2021 Drug and Biological HCPCS code application review cycle. Each summary includes: • Application number; • Topic/Issue; • Summary of the applicant's request as written by the applicant with occasional minor, non-substantive editorial changes made by CMS; • CMS' final or preliminary coding decision; and • Effective date of any coding action which, for the purpose of this publication, refers to the date the code is first available to be billed on claims. The HCPCS coding decisions below will also be included in the July 2021 HCPCS Quarterly Update, pending publication by CMS in the coming weeks at: https://www.cms.gov/Medicare/Coding/HCPCSReleaseCodeSets/Alpha-Numer ic-HCPCS. Request # 20.125 Topic/Issue Request to establish a new Level II HCPCS code to identify Fensolvi (leuprolide acetate) for injectable suspension for subcutaneous use. Applicant's suggested language: JXXXX “Injection, leuprolide acetate (Fensolvi), 45 mg, for subcutaneous use.” Applicant’s Summary Fensolvi is a sterile polymeric matrix formulation of leuprolide acetate that is administered subcutaneously by a healthcare professional for the treatment of pediatric patients 2 years of age and older with central precocious puberty. Leuprolide acetate is a synthetic nonapeptide analog of naturally occurring gonadotropin-releasing hormone that possesses greater potency than the natural hormone. Fensolvi is supplied in a kit that includes 2 prefilled syringes; 1 contains 45 mg of lyophilized leuprolide acetate powder and the other contains the in-situ polymeric gel delivery system.
    [Show full text]
  • Macrophage Colony Stimulation Factor for Relapsed/Refractory High-Risk Neuroblastoma
    HEALTH TECHNOLOGY BRIEFING FEBRUARY 2020 Naxitamab in combination with granulocyte- macrophage colony stimulation factor for relapsed/refractory high-risk neuroblastoma NIHRIO ID 20601 NICE ID 10208 Developer/Company Y-mAbs UKPS ID Not available Therapeutics Licensing and market Currently in phase II clinical trials. availability plans SUMMARY Naxitamab in combination with granulocyte macrophage colony stimulation (GM-CSF) is in clinical development for the treatment of patients with relapsed/refractory high risk neuroblastoma. Neuroblastoma is a rare cancer that mostly affects babies and young children. It develops from specialised nerve cells (neuroblasts) in the foetus that do not become mature nerve cells. Instead, they continue to grow and divide becoming cancerous. Relapsed or refractory high risk neuroblastoma has significant effect on children, young people and their families and carers. Existing treatments and procedures for neuroblastoma are painful and debilitating, with severe and long- lasting side effects. Naxitamab is a type of protein that has been designed to recognise and attach to a specific structure called GD2 that is present in high amounts on the surface of neuroblastoma cells, but not normal cells. Naxitamab attaches to the neuroblastoma cells and activates the immune system, which then kills the cancer cells. If licensed, naxitamab in combination with GM-CSF may provide a treatment option for patients with relapsed/refractory high-risk neuroblastoma. This briefing reflects the evidence available at the time of writing and a limited literature search. It is not intended to be a definitive statement on the safety, efficacy or effectiveness of the health technology covered and should not be used for commercial purposes or commissioning without additional information.
    [Show full text]
  • What Is Naxitamab?
    Home in time for supper: Humanized Anti-GD2 antibody in the outpatient setting Melanie Cohen, RN, BSN, CPHON and Katherine Alessi, RN, BSN, CPHON Pediatric Ambulatory Care Center; New York, New York What is Naxitamab? Sample Schedule Standard Operating Naxitamab (hu3F8) is a humanized monoclonal antibody that Days 1,3,5: Monday/Wednesday/Friday* Procedures (SOPs) adheres to GD2 on neuroblastoma cells. Naxitamab has shown 0800: Room/emergency equipment set-up stronger binding to GD2 than other known anti-GD2 antibodies. A specific set of practices initiated and followed 0815: Patient arrives to clinic, parent/patient education, obtain vital signs, when particular circumstances arise Potential advantages over murine antibodies such confirm/obtain IV access, draw labs as mu3F8 0900: Administer SQ GM-CSF (Sargramostim)500µg/m2/day Infusion Hypertension Allergic reaction 1. Low immunogenicity allowing repeat 0915: Premedications (analgesic, antihistamines, antipyretic, etc.) Onset of pain Hypotension Significant reaction treatments 2. Improved antibody-dependent cell-mediated 0930: Emergency medication preparation, history & physical by Nurse Practitioner cytotoxicity (ADCC) potency 1015: Begin Naxitamab infusion over ~35min 3. Longer serum half-life reducing the necessity 1100: Post Naxitamab monitoring -pain, hyper/hypotension, tachycardia, fever, hives, of daily infusions Response by MIBG imaging 4. Reduction of pain side effects, anaphylaxis nausea/vomiting, respiratory distress; vital signs q 1hr or as clinically indicated and anaphylactoid reactions as well as 1400: Discharge; instruction to patient/family 3 yr old girl (primary refractory) before and after 2 cycles immune complex disease Y-mAbs Therapeutics, Inc. *example schedule of Naxitamab/GM-CSF Phase II Study Design Side Effects Given in patients with high risk neuroblastoma.
    [Show full text]