US FDA sets PDUFA date for SCENESSE®

Priority Review granted for innovative drug, PDUFA date 8 July 2019

 

Executive summary

  • New Drug Application submitted in accordance with section 505(b) of the Federal Food, Drug, and Cosmetic Act, filed in accordance with 21 CFR 314.101(a)
  • Scientific exchange between FDA and CLINUVEL continues under Priority Review
  • FDA expects to communicate labelling and post-marketing commitments – if needed – by 8 April 2019
  • FDA does not plan to hold advisory committee meeting

 

Melbourne, Australia 10 January 2019

 

CLINUVEL PHARMACEUTICALS LTD today announced that the US Food and Drug Administration (FDA) Division of Dermatology and Dental Products has set a Prescription Drug User Fee Act (PDUFA) date of 8 July 2019. A New Drug Application (NDA) for the use of SCENESSE® (afamelanotide 16mg) in the prevention of phototoxicity and anaphylactoid reactions in adult patients with erythropoietic protoporphyria (EPP) had been submitted under Section 505b of the Federal Food, Drug, and Cosmetic Act.


REVIEW UNDER PDUFA VI

PDUFA establishes target dates for review of NDAs by the FDA. SCENESSE® will be evaluated under PDUFA VI, reauthorised by the Food and Drug Administration Reauthorization Act of 2017, as a Priority Review.

Following an assessment under 21 CRF 314.101(a) for NDA completeness, the FDA review then assesses the risk-benefit profile of the product for the intended patient population. A scientific exchange is expected between the FDA and CLINUVEL during the final review process, with answers provided to regulatory questions on all aspects of the technical dossier. The FDA has advised that it does not intend to hold an advisory committee meeting during the final review of the SCENESSE® NDA, with proposed labelling and post-marketing requirements – if needed – to be communicated to CLINUVEL by 8 April 2019.


COMMENTARY

“Acceptance of this NDA is the result of patience and hard work from the CLINUVEL team and medical community, bringing us a significant step closer to making SCENESSE® available for US EPP patients,” CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said. “We are pleased with the work of the FDA to date and, contrary to our own expectations, the agency has stated that it does not intend to hold an advisory committee meeting for further review of the product.

“We will continue the open and constructive dialogue with the FDA in the coming weeks and months to work towards a positive and landmark outcome for EPP patients on 8 July 2019,” Dr Wright said.


SCENESSE® FOR EPP

SCENESSE® is a controlled release injectable implant containing the novel active ingredient afamelanotide. The drug was developed as a first-line treatment for patients with EPP, a rare genetic metabolic disorder which causes phototoxicity and anaphylactoid reactions when patients expose their skin to light. CLINUVEL conducted five clinical trials of SCENESSE® in EPP. Two randomised, placebo-controlled clinical trials of SCENESSE® conducted at US EPP expert centres showed the drug enabled patients to increase the amount of time spent outside without experiencing phototoxicity and improved patient quality of life.

SCENESSE® was granted orphan drug designation by the FDA in 2008. In July 2016 the FDA, having assessed the clinical data package for the main EPP studies, advised that the data were ready for NDA submission, and in November 2016 a pre-NDA meeting was held. In October 2016, the FDA organised a first Scientific Workshop on EPP as part of a pilot scheme to involve patients and their families in the scientific review of disease and treatment solutions. On 22 June 2018 CLINUVEL filed the final module of the NDA for SCENESSE® under “rolling review”. Additional data were submitted in response to questions from the FDA during the preliminary review period.

SCENESSE® was approved for the prevention of phototoxicity in adult patients with EPP in Europe in 2014.1 CLINUVEL seeks US regulatory approval for the same treatment dose and regimen in the United States as is currently approved in the European Union, where SCENESSE® is prescribed to EPP patients by clinical experts at specialised treatment centres. There are currently no therapies approved for EPP patients in the US.

– End –

 

1 SCENESSE® (afamelanotide 16mg) is approved in Europe as an orphan medicinal product for the prevention of phototoxicity in adult patients with EPP. Information on the product can be found on CLINUVEL’s website at www.clinuvel.com.


About CLINUVEL PHARMACEUTICALS LIMITED

CLINUVEL PHARMACEUTICALS LTD (ASX: CUV; NASDAQ INTERNATIONAL DESIGNATION ADR: CLVLY; XETRA-DAX: UR9) is a global biopharmaceutical company focused on developing and delivering treatments for patients with a range of severe genetic and skin disorders. As pioneers in photomedicine and understanding the interaction of light and human biology, CLINUVEL’s research and development has led to innovative treatments for patient populations with a clinical need for photoprotection and repigmentation. These patient groups range in size from 5,000 to 45 million worldwide. CLINUVEL’s lead compound, SCENESSE® (afamelanotide 16mg), was approved by the European Commission in 2014 for the prevention of phototoxicity (anaphylactoid reactions and burns) in adult patients with erythropoietic protoporphyria (EPP). More information on EPP can be found at http://www.epp.care.

Headquartered in Melbourne, Australia, CLINUVEL has operations in Europe, Switzerland, the US and Singapore, with the UK acting as the EU distribution centre.

For more information go to http://www.clinuvel.com.

SCENESSE® is a registered trademark of CLINUVEL PHARMACEUTICALS LTD.


Investor enquiries

InvestorRelations@clinuvel.com

Media enquiries

Europe: Lachlan Hay, CLINUVEL (UK) LTD. +44 1372 860 765 Lachlan.Hay@clinuvel.com

USA: Terri Clevenger, Continuum Health Communications, +1 (203) 227-0209, tclevenger@continuumhealthcom.com

 

 

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