CLINUVEL to Launch SCENESSE® in USA in April 23 March 2020
CLINUVEL PHARMACEUTICALS LTD today announced plans to launch its novel drug SCENESSE® (afamelanotide) in the USA, with the first patient to be treated after 15 April 2020. SCENESSE® was approved by the US Food and Drug Administration in October 2019 to increase pain free light exposure in adult patients with a history of phototoxic reactions from the rare metabolic disorder erythropoietic protoporphyria (EPP).
USA DISTRIBUTION SCENESSE®
The distribution of SCENESSE® is planned in three phases. During Phase I, three selected hospitals and medical centers will be able to provide treatment to EPP patients. For Phase II, Medicare-Medicaid will need to complete its review of the SCENESSE® dossier and provide an opinion on National and Local Coverage for the treatment, as to be confirmed by the Centers for Medicare & Medicaid Services. The last phase will consist of direct distribution of the drug to a target maximum of 30 centers who will be trained and accredited by CLINUVEL.
INSURANCE COVERAGE SCENESSE®
During the first phase of distribution commencing 15 April 2020, patients will be able to receive treatment under Prior Authorization [PA]. PA is a decision by a payor that a healthcare service, treatment plan, prescription drug, or durable medical equipment is medically necessary and is included in a member’s (patient’s) coverage.
Clinuvel Pharmaceuticals is the leading photoprotective pharmaceutical company. Headquartered in Melbourne, Australia with offices in Baar, Switzerland, Singapore and Los Angeles Clinuvel trades as an ADR in the US. Our Work:
Source: http://www.clinuvel.com/en/our-work Clinuvel is developing new drugs to improve the lives of individuals with severe skin disorders. Our lead technologies, SCENESSE® (afamelanotide 16mg) and CUV9900, are from a new class of drugs, known as melanocortins, which are based on the naturally occurring hormone α-MSH. Mission Statement: Source: http://www.clinuvel.com/en/mission-statement Clinuvel’s Mission is to provide treatment for patients who are most severely affected by light/UV and depigmentation disorders. Clinuvel develops pharmaceutical products, which belong to the family of melanocortins, to be used as prescriptive therapies.
Head Office: Source: http://www.clinuvel.com/en/contact Clinuvel Pharmaceuticals Limited,
190 Queen Street,
Tel: +61 3 9660 4900
Fax: +61 3 9660 4999
The technology behind Clinuvel’s lead drug, SCENESSE® (afamelanotide 16mg), has been in research and development since the 1980s when a small team of scientists set out to investigate methods of preventing skin cancer in humans. Clinuvel licensed this technology and has spent over A$20million optimising the chemistry of afamelanotide (the active ingredient in SCENESSE®) and developing a proprietary, controlled-release implant formulation.
Since late 2005, Clinuvel has focused on developing SCENESSE® as a dermatological drug for individuals at greatest risk from visible and ultraviolet (UV) light and as a repigmentation therapy. The company has a particular focus on diseases where no existing preventative treatment exists.
Clinuvel commenced a clinical program in 2006 which focused on the rare disease erythropoietic protoporphyria (EPP), a metabolic disorder which causes painful burning reactions under the skin after even brief exposure to sunlight. The goal of Clinuvel’s EPP program is to prove that SCENESSE® can provide patients with EPP protection from sunlight by reducing the incidence and severity of EPP reactions. The ultimate objective is to provide a better quality of life for patients. Since the start of the EPP program, the company has involved more than 250 individuals with EPP in its global clinical trials. Between 2005 and today, clinical programs have also been conducted for individuals diagnosed with a range of disorders, including polymorphous light eruption, solar urticaria and organ transplant recipients who are susceptible to an extremely high rate of skin cancers.
In late 2009, Clinuvel announced that it had expanded its pipeline to include a second drug candidate, named CUV9900. Based on the same technology as SCENESSE®, CUV9900 is still in early stage development.
2010 was a pivotal year for Clinuvel. In May 2010, SCENESSE® became the first dermatological drug listed under the Italian 648/96 law, prior to its formal approval anywhere in the world. This listing allows Italian physicians to prescribe SCENESSE® to individuals with EPP with the cost of the drug reimbursed by the Italian National Health System (Sistema Sanitario Nazionale, SSN).
Later in 2010, Clinuvel also announced that it would expand its program for SCENESSE® to investigate the drug’s ability to repigment skin in the common pigmentation disorder vitiligo. Following regulatory and ethical approvals, Clinuvel’s International SCENESSE® Pilot Repigmentation Evaluation (INSPIRE) program commenced patient recruitment in 2011. A Phase II study was completed in the USA in 2013, showing that the drug had a good safety profile in vitiligo and may assist in faster repigmentation in patients when used in combination with narrowband UVB phototherapy. A Phase IIb study of the drug in vitiligo commenced in 2014 in Singapore.
In February 2012 Clinuvel lodged its first marketing authorisation application for SCENESSE® for EPP with the European Medicines Agency. In December 2014 the European Commission approved SCENESSE® for the prevention of phototoxicity in adult EPP patients.
As of December 2014, more than 900 individuals have been administered afamelanotide in Clinuvel’s clinical trials program and through compassionate use and special access schemes. To date, the drug has maintained a good safety profile in clinical trials.
SCENESSE® attains historic breakthrough European Marketing Authorisation
European Medicines Agency CHMP approves novel drug for erythropoietic protoporphyria patients under exceptional circumstances (ART 14 (8) OF REGULATION (EC) NO 726/2004)
- SCENESSE® attains an historic breakthrough EMA approval for adult EPP patients extremely intolerant of light and UV
Marketing authorisation in 31 European States
Australian listed Clinuvel (ASX: CUV) has taken SCENESSE® from discovery to commercialisation
SCENESSE® as the first drug for which patients’ and physicians’ clinical experiences were integrated EMA’s formal decision process
Investor telephone conference-call Wednesday 29 October: 1800 Melbourne = 0900 Zurich = 0300 New York = 1500 Singapore. Details will be released to the ASX on Tuesday October 28.
Clinuvel Pharmaceuticals Limited (ASX: CUV; XETRA-DAX: UR9; ADR: CLVLY) is proud to announce that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has voted in favour of marketing authorisation (MA) of Clinuvel’s drug SCENESSE® (afamelanotide 16mg) for adult patients with erythropoietic protoporphyria (EPP).
SCENESSE® is a first-in-class drug (belonging to a new drug category). EPP is a debilitating, rare genetic disorder clinically regarded as extreme intolerance to light and UV (phototoxicity). An estimated 10,000 EPP patients are affected worldwide, 45% of whom live within Europe.
Clinuvel conducted five trials testing SCENESSE® in approximately 350 adult EPP patients across Australia, Europe and the USA. The first EPP patient received the drug in 2006. Clinuvel submitted its dossier for EMA evaluation on 6 February 2012.
In September 2014, it was announced that the EMA - for the first time in its history - was incorporating patients’ and physicians’ clinical experiences with SCENESSE® in the CHMP’s decision process.
On 23 October 2014 in its plenary session the CHMP voted in favour of marketing authorisation of SCENESSE®. This decision takes into account the challenges and limitations of conducting clinical trials in rare and severe disorders and allows access to treatment for patients without alternative medcine.1 Marketing authorisation is given for distribution of SCENESSE® across 31 European states.2 The CHMP’s decision will now be sent to the European Commission for formal ratification, expected within 67 days.
Clinuvel and the EMA have agreed to a comprehensive post-authorisation pharmacovigilance plan to monitor patients’ safety long term. SCENESSE® will be distributed through academic and specialised centres.
“On behalf of the Board, I congratulate shareholders and patients, experts in the field and Clinuvel’s teams around the world,” Clinuvel’s Chair, Stan McLiesh said. “I have witnessed from our staff a decade long dedication and an enormous zeal which has come together for patients and all stakeholders in a marvellous outcome, one rarely accomplished in pharmaceutical development and which is now unique to Australia.”
“Medical innovation requires an exceptional focus with a consistent strategy, passionate team and long term trust from patients, expert physicians and regulatory authorities worldwide,” Clinuvel’s CEO, Dr Philippe Wolgen said.
“Today I am excited but mostly proud that Clinuvel overcame a number of hurdles from having identified an unmet clinical need and taken a molecule from discovery to final commercial product. I congratulate our Acting Chief Scientific Officer Dr Wright and his entire team for the extraordinary achievement.”
“This outcome greatly impacts Clinuvel, our further operations and intrinsic value. The immediate goal is to distribute the drug to European patients in the coming months, while we owe it to our American and Asia-Pacific patient community to accelerate the regulatory process for them to gain access to SCENESSE®,” Dr Wolgen said.
FDA APPROVAL PROCESS FDA decision is expected by October 8, 2019
The FDA has recognised that afamelanotide meets an unmet clinical need and treats a severe genetic condition for patients who are life-long deprived of light. SCENESSE®
was granted orphan drug designation by the FDA in 2008 for the treatment of EPP patients and Fast Track Designation (FTD) in 2016, allowing for a ‘rolling review’ of the NDA. The orphan drug designation provides R&D and review incentives for drugs which may not otherwise be commercially viable to develop, while FTD assists to ensure that innovative drugs reach the patient population earlier than would be the case during a standard review process. The ‘rolling review’ under the FTD allows the FDA to start the review of the scientific dossier only when all modules have been submitted and have passed formal validation, a two-month process after submission of the final NDA module.
CLINUVEL obtained a positive FDA answer on acceptance of the current safety data, as the FDA issued a carcinogenicity waiver in 2017.
This year CLINUVEL applied for a Priority Review which would secure a maximum review period of six months, compared to the standard ten months. It is expected that the FDA will answer the request for Priority Review at the start of the review period.
The FDA has initiated a regulatory pilot scheme to involve patients in explaining the impact of diseases on their lives. The Division for Dental and Dermatology Products hosted a scientific workshop on EPP in October 2016.
The NDA dossier is expected to be lodged in Q4 2017 or Q1 2018 which could lead to approval of Scenesse Q3 2018.
Following the approval of Clinuvel’s lead product SCENESSE® (afamelanotide 16mg), the Company is actively pursuing an expanded pipeline of products from the melanocortin family, a class of drugs that includes afamelanotide. Two novel molecules have been announced: CUV9900 and VLRX001.
CUV9900 is an alpha-Melanocyte Stimulating Hormone (alpha-MSH) analogue. Initial research has shown that CUV9900 is a potent skin protectant. Clinuvel intends to launch CUV9900 in various new formulations. Formulation work has commenced and Clinuvel expects the first formulations to be available for clinical testing after the commercialisation of SCENESSE® in Europe.
VLRX001 is an addition to the family of melanocortin analogues which provoke increased and prolonged cellular activity. It contains a specific peptide sequence, designed to make it less susceptible to degradation than physiologic (natural) alpha-melanocyte stimulating hormone (α-MSH).
The VLRX001 development work was undertaken through Clinuvel’s majority owned Singaporean subsidiary, VALLAURIX and has leveraged the knowledge gained from long term experience with the clinical use of SCENESSE®.
Formulation work will focus on the development of VLRX001 for topical self-administration by patients. The transdermal product will initially be evaluated as adjuvant maintenance therapy in the depigmentation disorder vitiligo.
Overall, the development of VLRX001 is part of Clinuvel’s life cycle management of its portfolio of products to ensure long term use and value of its technology and assets. Clinuvel will hold all rights to the final product and indications through VALLAURIX.
UPDATED: NOV. 2017