Date: 2016-10-13
Type of information: Submission of a Market Application in the US
Product name: Scenesse®
Compound: afamelanotide
Therapeutic area: Rare diseases - Genetic diseases
Action mechanism: peptide. The active substance of Scenesse® is afamelanotide, a synthetic analogue of the physiologically occurring alpha-melanocyte stimulating hormone that acts as a melanocortin-1 receptor agonist thereby stimulating melanogenesis and emollients and protectives (D02BB02). The increased melanogenesis results in increased pigmentation of the skin.
Company: Clinuvel UK (UK) Clinuvel (Australia)
Disease: erythropoietic protoporphyria
Latest news: * On October 13, 2016, Clinuvel Pharmaceuticals announced that it will meet with the FDA on 7 November to * On May 18, 2016, Clinuvel Pharmaceuticals announced an update on the company’s post-authorisation distribution of Scenesse® across Europe for adult patients with the rare disorder erythropoietic protoporphyria. * On February 16, 2016, Clinuvel Pharmaceuticals announced an update on the commercialisation of Scenesse® (afamelanotide 16mg) in adult patients with erythropoietic protoporphyria (EPP) in Europe. Clinuvel has been working towards finalising distribution infrastructure, with a focus on a Risk Management Plan (RMP) and pharmacovigilance in accordance to EU legislation to undertake the long term follow up of EPP patients receiving Scenesse®. Patients will be treated in accordance with a Post-Authorisation Safety (PASS) Protocol. Payors across Europe acknowledge the need to treat EPP, a rare genetic disorder which is regarded as the most extreme form of phototoxicity, an anaphylactoid reaction to light and UV. Advisory bodies and healthcare organisations in a number of European countries are currently inviting patient representatives and expert physicians to closed workshops to assess the burden of EPP and the proposed access to Scenesse® as part of the process of determining a reimbursement price under current national healthcare provisions. Factors such as development, manufacturing and distribution costs, as well as specific expenditures related to the long term follow up of patients determine the pricing of the product in Europe and other regions. Discussions are ongoing and final pricing is not yet established. Clinuvel expects the first patients to receive treatment with Scenesse® pending regulatory requirements and pricing agreements in individual European countries. It is anticipated that, in the coming weeks, various European authorities will publish details relevant to market access and distribution. * On 23 October 2014, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for Scenesse®, 16 mg, implant, intended for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). Scenesse was designated as an orphan medicinal product on 8 May 2008.
formally discuss lodging its new drug application (NDA) for Scenesse® (afamelanotide 16mg). The pre-NDA meeting will focus on finalising requirements for filing Scenesse® with the FDA for the treatment of adult patients with erythropoietic protoporphyria (EPP). The pre-NDA meeting, with the FDA’s Division of Dermatology and Dental Products (DDDP), is part of ongoing dialogue between Clinuvel and the FDA since Clinuvel commenced a clinical program for EPP in 2006.
Earlier this year the FDA granted Scenesse® Fast Track designation, enabling a rolling review of the NDA dossier. The FDA also completed an initial review of Clinuvel’s clinical data package, deeming it satisfactory and sufficient for NDA submission. On 24 October the DDDP will host an EPP Workshop to obtain the patients’ and physicians’ perspective on certain disease areas, including the effectiveness of treatments. Scenesse® is the first treatment ever evaluated in contemporary clinical trials for EPP. The drug provides photoprotection to EPP patients who suffer from acute reactions to visible light and sun (phototoxicity). In 2014 Scenesse® was approved in Europe for the prevention of phototoxicity in adult EPP patients, with the drug now prescribed in a number of European countries.
Clinuvel conducted clinical trials of Scenesse® in EPP from 2006 to 2013. During this time the company evaluated the treatment in 352 EPP patients. After completion of the clinical trial program, approximately 130 patients across eight countries (Australia, Austria, France, Germany, Italy, Sweden, Switzerland and The Netherlands) received Scenesse® free of charge. In total 601 implant injections were provided to patients free of charge under compassionate use schemes. The development program and the following compassionate use program was supported entirely by equity funding.
Subsidised access to Scenesse® treatment, prior to the drug’s formal commercial approval, was made possible in
both Italy and Switzerland from 2010 and 2012 respectively. These two programs enabled 115 EPP patients to
benefit from treatment with Scenesse® totalling more than 200 patient years of cumulative care. Clinuvel is no
longer in the position to provide access to Scenesse® on a subsidised basis.
The European Medicines Agency (EMA) granted Scenesse® marketing authorisation under a strict risk management plan, comprising a Post-Authorisation Safety Study (PASS) as well as a Retrospective Chart Review (RCR) to follow up those who do not consent to participate in the PASS. The process of regulatory drug release and approval of the pharmacovigilance systems required a further 18 months of in-depth discussion with the EMA. The EMA’s pharmacovigilance subcommittee (PRAC) formally endorsed the last of these protocols on April 14, 2016.
The final regulatory and clinical steps prior to the start of the treatment are a review of the PASS and RCR
protocols by some of the local ethics committees and the approval of patient and physician educational materials
by various National Competent Authorities.
Scenesse® is only being made available to EPP patients through Porphyria Expert Centres, which require
training and accreditation by Clinuvel. In Germany it is anticipated that the first year three Porphyria Expert Centres – in Berlin, Chemnitz, and Düsseldorf – will be able to prescribe the treatment. The German national authorities have released their first assessment of the use of Scenesse® in EPP. In parallel, 13 German insurance firms have agreed to make Scenesse® available to EPP patients. Discussions are underway with authorities in nine countries to make Scenesse® available, with further submissions anticipated in 2016.
* On March 24, 2016, Clinuvel Pharmaceuticals announced that the National Institute for Health and Care Excellence (NICE) has held a public workshop to scope the benefits and costs of Scenesse® (afamelanotide 16mg) in the treatment of adult patients with erythropoietic protoporphyria. This workshop is one of the last steps prior to national commissioning of the treatment by the National Health Service (NHS) England. The company was invited alongside representatives of the EPP patient community, clinical experts and scientists to discuss the benefits of Scenesse® and its characterisation as a Highly Specialised Technology (HST) in England. The meeting included a review of the specific burden of the disease on patients’ lives, the number of treatment centres in the UK, on patients eligible for treatment and the lack of a standard of care. NICE’s HST team will use feedback from the workshop to finalise a remit for final assessment by the Department of Health.
* On March 15, 2016, Clinuvel Pharmaceuticals announced that its European commercial team is conducting site training and accreditation at the first of the European expert porphyria centres. Following training these centres of expertise will be accredited to prescribe Clinuvel’s Scenesse® to adult patients diagnosed with erythropoietic protoporphyria. Clinuvel has implemented a structured training program for clinical staff in the multidisciplinary expert porphyria treatment centres throughout Europe. The company will ensure compliance with a treatment protocol as part of a PASS. Clinuvel will track and monitor pharmacovigilance, drug accountability, use of the European EEP Disease Registry (EEDR), and correct handling of the Scenesse® drug product. Contracts with all expert centres aim to facilitate compliance with the PASS and continuous long-term care of EPP patients.
The benefits with Scenesse® are a decrease in light sensitivity, which translates into a limited increase in time EPP patients may spend in daylight or sunlight. Patients report an improved quality of life with increased time spent outside (commuting, work, leisure/sport, social). The most common side effects are headache, nausea, nasopharyngitis, migraine, abdominal pain, fatigue, lethargy and somnolence. A pharmacovigilance plan for Scenesse® will be implemented as part of the marketing authorisation.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization: 2014-12-21
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2008-05-08
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
