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Scenesse EPAR Assessment Report 23 October 2014 EMA/CHMP/709396/2014 Rev.1 Committee for Medicinal Products for Human Use (CHMP) Assessment report SCENESSE International non-proprietary name: afamelanotide Procedure No. EMEA/H/C/002548/0000 Note Assessment report as adopted by the CHMP with all information of a commercially confidential nature deleted. 30 Churchill Place ● Canary Wharf ● London E14 5EU ● United Kingdom Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5520 Send a question via our website www.ema.europa.eu/contact An agency of the European Union © European Medicines Agency, 2015. Reproduction is authorised provided the source is acknowledged. Administrative information Name of the medicinal product: Scenesse Applicant: Clinuvel (UK) Limited c/o Reed Smith Broadgate Tower, Third Floor 20 Primrose Street London EC2A 2RS United Kingdom Active substance: afamelanotide International Nonproprietary Name/Common afamelanotide Name: Pharmaco-therapeutic group Emollients and protectives (ATC Code): (D02BB02) prevention of phototoxicity in adult patients Therapeutic indication: with erythropoietic protoporphyria (EPP) Pharmaceutical form: Implant Strength: 16 mg Route of administration: Subcutaneous use Packaging: vial (glass) Package size: 1 vial Assessment report EMA/CHMP/601433/2014 Page 2/107 Table of contents 1. Background information on the procedure .............................................. 7 1.1. Submission of the dossier ...................................................................................... 7 1.2. Manufacturers ...................................................................................................... 8 1.3. Steps taken for the assessment of the product ......................................................... 9 2. Scientific discussion .............................................................................. 10 2.1. Introduction....................................................................................................... 10 2.2. Quality aspects .................................................................................................. 14 2.2.1. Introduction .................................................................................................... 14 2.2.2. Active substance ............................................................................................. 15 2.2.3. Finished medicinal product ................................................................................ 17 2.2.4. Discussion on chemical, and pharmaceutical aspects ............................................ 19 2.2.5. Conclusions on the chemical, pharmaceutical and biological aspects ...................... 20 2.2.6. Recommendations for future quality development ............................................... 20 2.3. Non-clinical aspects ............................................................................................ 21 2.3.1. Introduction .................................................................................................... 21 2.3.2. Pharmacology ................................................................................................. 21 2.3.3. Pharmacokinetics............................................................................................. 28 2.3.4. Toxicology ...................................................................................................... 29 2.3.5. Ecotoxicity/environmental risk assessment ......................................................... 35 2.3.6. Discussion on non-clinical aspects...................................................................... 35 2.3.7. Conclusion on the non-clinical aspects ................................................................ 38 2.4. Clinical aspects .................................................................................................. 38 2.4.1. Introduction .................................................................................................... 39 2.4.2. Pharmacokinetics............................................................................................. 40 2.4.3. Pharmacodynamics .......................................................................................... 42 2.4.4. Discussion on clinical pharmacology ................................................................... 45 2.4.5. Conclusions on clinical pharmacology ................................................................. 46 2.5. Clinical efficacy .................................................................................................. 47 2.5.1. Dose response study ........................................................................................ 47 2.5.2. Main study ...................................................................................................... 47 2.5.3. Discussion on clinical efficacy ............................................................................ 83 2.5.4. Conclusions on the clinical efficacy ..................................................................... 90 2.6. Clinical safety .................................................................................................... 91 2.6.1. Discussion on clinical safety .............................................................................. 94 2.6.2. Conclusions on the clinical safety ....................................................................... 96 2.7. Pharmacovigilance .............................................................................................. 97 2.8. Risk Management Plan ........................................................................................ 97 Paediatric population .............................................................................. 100 2.9. Product information .......................................................................................... 101 2.9.1. User consultation ........................................................................................... 101 Assessment report EMA/CHMP/601433/2014 Page 3/107 3. Benefit-Risk Balance............................................................................ 101 4. Recommendations ............................................................................... 104 Assessment report EMA/CHMP/601433/2014 Page 4/107 List of abbreviations ACTH adrenocorticotrophic hormone AGRP agouti-related protein ASMF active substance master file ASP agouti signalling protein α-MSH alpha-melanocyte stimulating hormone (melanotropin) cAMP 3’-5’-cyclic adenosine monophosphate CNS central nervous system EEG electroencephalogram EPP erythropoietic protoporphyria ERA environmental risk assessment F female FFA free fatty acids FTIR Fourier transform infrared spectroscopy GC gas chromatography GD gestation day GPC gel permeation chromatography HPA Hypothalamic-Pituitary-Adrenal axis HPLC high performance liquid chromatography ICH International Conference of Harmonization IL intermediary lobe ip intraperitoneal KF Karl Fischer LH Lister Hooded (rat strain) M male MC1-R melanocortin 1 receptor MCV mean cell volume MED minimal erythema dose MTD maximum tolerated dose MS/MS tandem mass spectrometry MPCE micronucleated polychromatic erythrocytes NCE normochromatic erythrocytes Assessment report EMA/CHMP/601433/2014 Page 5/107 NMR nuclear magnetic resonance NZW New Zealand White PEC predicted environmental concentration PCE polychromatic erythrocytes Ph. Eur. European Pharmacopoeia PK pharmacokinetics PLG poly(DL-lactide-co-glycolide) POMC pro-opiomelanocortin po per os RH relative humidity sc subcutaneous SD Sprague Dawley (rat strain) SmPC summary of product characteristics TK toxicokinetics UVR ultra violet radiation Assessment report EMA/CHMP/601433/2014 Page 6/107 1. Background information on the procedure 1.1. Submission of the dossier The applicant Clinuvel (UK) Limited submitted on 3 February 2012 an application for Marketing Authorisation to the European Medicines Agency (EMA) for SCENESSE, through the centralised procedure falling within the Article 3(1) and point 4 of Annex of Regulation (EC) No 726/2004. The eligibility to the centralised procedure was agreed upon by the EMA/CHMP on 14 April 2011. Scenesse, was designated as an orphan medicinal product EU/3/08/541 on 8 May 2008 in the following indication: Treatment of erythropoietic protoporphyria. The applicant applied for the following indication: prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). Following the CHMP positive opinion on this marketing authorisation, the Committee for Orphan Medicinal Products (COMP) reviewed the designation of Scenesse as an orphan medicinal product in the approved indication. The outcome of the COMP review can be found on the Agency's website: ema.europa.eu/Find medicine/Rare disease designations. The legal basis for this application refers to: Article 8.3 of Directive 2001/83/EC - complete and independent application. The applicant indicated that afamelanotide was to be considered as a new active substance. The application submitted is composed of administrative information, complete quality data, non-clinical and clinical data based on applicants’ own tests and studies. Information on Paediatric requirements Pursuant to Article 7 of Regulation (EC) No 1901/2006, the application included an EMA Decision P/292/2011 on the agreement of a paediatric investigation plan (PIP). At the time of submission of the application, the PIP P/292/2011 was not yet completed as some measures were deferred. A waiver was granted for all subsets of the paediatric
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