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Luxturna, INN-Voretigene Neparvovec 20 September 2018 EMA/CHMP/700911/2018 Committee for Medicinal Products for Human Use (CHMP) Assessment report Luxturna International non-proprietary name: voretigene neparvovec Procedure No. EMEA/H/C/004451/0000 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 5555 Send a question via our website www.ema.europa.eu/contact An agency of the European Union © European Medicines Agency, 2019. Reproduction is authorised provided the source is acknowledged. Table of contents 1. Background information on the procedure .............................................. 6 1.1. Submission of the dossier ...................................................................................... 6 1.2. Steps taken for the assessment of the product ......................................................... 7 2. Scientific discussion ................................................................................ 9 2.1. Problem statement ............................................................................................... 9 2.1.1. Disease or condition ........................................................................................... 9 2.1.2. Epidemiology .................................................................................................... 9 2.1.3. Biologic features ................................................................................................ 9 2.1.4. Clinical presentation and diagnosis .................................................................... 10 2.1.5. Management ................................................................................................... 10 2.2. Quality aspects .................................................................................................. 11 2.2.1. Introduction .................................................................................................... 11 2.2.2. Active Substance ............................................................................................. 11 2.2.3. Finished Medicinal Product ................................................................................ 15 2.2.4. Discussion on chemical, pharmaceutical and biological aspects .............................. 17 2.2.5. Conclusions on the chemical, pharmaceutical and biological aspects ...................... 18 2.2.6. Recommendation(s) for future quality development ............................................. 18 2.3. Non-clinical aspects ............................................................................................ 18 2.3.1. Introduction .................................................................................................... 18 2.3.2. Pharmacology ................................................................................................. 18 2.3.3. Pharmacokinetics............................................................................................. 20 2.3.4. Toxicology ...................................................................................................... 20 2.3.5. Ecotoxicity/environmental risk assessment ......................................................... 20 2.3.6. Discussion on the non-clinical aspects ................................................................ 21 2.3.7. Conclusion on the non-clinical aspects ................................................................ 23 2.4. Clinical aspects .................................................................................................. 23 2.4.1. Introduction .................................................................................................... 23 2.4.2. Pharmacokinetics............................................................................................. 23 2.4.3. Pharmacodynamics .......................................................................................... 23 2.4.4. Discussion on clinical pharmacology ................................................................... 24 2.4.5. Conclusions on clinical pharmacology ................................................................. 24 2.5. Clinical efficacy .................................................................................................. 24 2.5.1. Dose response study ........................................................................................ 24 2.5.2. Main study ...................................................................................................... 25 2.5.3. Discussion on clinical efficacy ............................................................................ 59 2.5.4. Conclusions on the clinical efficacy ..................................................................... 64 2.6. Clinical safety .................................................................................................... 64 2.6.1. Discussion on clinical safety .............................................................................. 75 2.6.2. Conclusions on the clinical safety ....................................................................... 76 2.7. Risk Management Plan ........................................................................................ 77 2.8. Pharmacovigilance .............................................................................................. 82 2.9. New Active Substance ......................................................................................... 82 2.10. Product information .......................................................................................... 82 Assessment report EMA/CHMP/700911/2018 Page 2/97 2.10.1. User consultation ........................................................................................... 82 2.10.2. Labelling exemptions ...................................................................................... 82 2.10.3. Additional monitoring ..................................................................................... 84 3. Benefit-Risk Balance.............................................................................. 84 3.1. Therapeutic Context ........................................................................................... 84 3.1.1. Disease or condition ......................................................................................... 84 3.1.2. Available therapies and unmet medical need ....................................................... 85 3.1.3. Main clinical studies ......................................................................................... 85 3.2. Favourable effects .............................................................................................. 85 3.3. Uncertainties and limitations about favourable effects ............................................. 86 3.4. Unfavourable effects ........................................................................................... 86 3.5. Uncertainties and limitations about unfavourable effects ......................................... 87 3.6. Effects Table ...................................................................................................... 89 3.7. Benefit-risk assessment and discussion ................................................................. 93 3.7.1. Importance of favourable and unfavourable effects .............................................. 93 3.7.2. Balance of benefits and risks ............................................................................. 93 3.7.3. Additional considerations on the benefit-risk balance ........................................... 93 3.8. Conclusions ....................................................................................................... 94 4. Recommendations ................................................................................. 94 Assessment report EMA/CHMP/700911/2018 Page 3/97 List of abbreviations Assessment report EMA/CHMP/700911/2018 Page 4/97 Assessment report EMA/CHMP/700911/2018 Page 5/97 1. Background information on the procedure 1.1. Submission of the dossier The applicant Spark Therapeutics Ireland Ltd submitted on 29 July 2017 an application for marketing authorisation to the European Medicines Agency (EMA) for Luxturna, through the centralised procedure falling within the Article 3(1) and point 1 of Annex of Regulation (EC) No 726/2004. The eligibility to the centralised procedure was agreed upon by the EMA/CHMP on 28 April 2016. Luxturna was designated as an orphan medicinal product on 02 April 2012 in the following condition ‘treatment of Leber's congenital amaurosis’ (EU/3/12/981), and on 28 July 2015 in the following condition ‘treatment of retinitis pigmentosa’ (EU/3/15/1518). The applicant applied for the following indication: Luxturna is indicated for the treatment of patients with vision loss due to Leber’s congenital amaurosis or retinitis pigmentosa inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations. The legal basis for this application refers to: Article 8.3 of Directive 2001/83/EC - complete and independent application. The applicant indicated that voretigene neparvovec was considered to be 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 and/or bibliographic literature substituting/supporting certain tests or studies. Information on Paediatric requirements
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