Rare Disease Report

Amicus Acquires Scioderm: To Begin Rolling NDA for Epidermolysis Bullosa Drug in 2016

AUGUST 30, 2015
James Radke, PhD
Amicus Therapeutics announced they plan to acquire 100% of Scioderm, the developers of the cream Zorblisa that is in late stage development for the treatment of epidermolysis bullosa—a rare, genetic, skin disorder.

The acquisition is a new approach by Amicus, which has been mostly focused on lysosomal diseases (Fabry, Pompe) until now. However, as John Crowley, CEO of Amicus explained in a conference call, Zorblisa has 5 things that Amicus was looking for to expand their pipeline. Zorlisa was associated with:
  1. A rare disease
  2. Devastating disorder
  3. First in class or best in class therapy
  4. Meaningful clinical benefit for patients
  5. Significant orphan patient population.

Regulatory Timeline

Zorblisa has begun enrolling patients for a phase 3 clinical trial. Based on the phase 2 data, the FDA has granted the drug a breakthrough therapy designation. Amicus plans to begin a rolling NDA later this year (and completed the second half of 2016). They also hope to obtain a pediatric priority review voucher if approved.

Business Potential

Amicus estimates that epidermolysis bullosa may represent a potential $1 billion+ global market opportunity. The current standard of care is palliative treatments, which cost $10,000 to $15,000 per month, and mainly consist of bandaging, treating the open wounds to prevent infection and trying to manage patients' pain. An estimated 30,000 to 40,000+ people are currently diagnosed with epidermolysis bullosa in major markets.

Clinical Trials

Zorblisa has completed its phase 2 study. In that study, patients given Zorblis (6% cream; n=12) had a median time to complete healing of 30 days compared to 91 days in the patients given placebo (n=17). A Phase 3 multi-center, randomized, double-blind, placebo-controlled study in the United States and Europe is currently underway and expected to support registration globally. The study is enrolling individuals who are 1 month and older with a diagnosis of simplex, recessive dystrophic, or junctional non-Herlitz epidermolysis bullosa who have at least 1 target wound present for 21 days or more. Half the patients receive Zorblisa cream and the other half receive placebo cream, applied topically once daily to the entire body for 90 days. The primary outcome measure is complete target wound closure within 2 months.

The Transaction

Amicus will pay Scioderm shareholders $229 million. Amicus will also pay up to $361 million to Scioderm shareholders upon achievement of certain clinical and regulatory milestones and $257 million upon achievement of certain sales milestones. If a Priority Review Voucher is obtained upon approval, Amicus will pay Scioderm shareholders the lesser of $100 million or 50% of the proceeds of such sale.

Epidermolysis Bullosa

Epidermolysis bullosa ( or EB) is a group of rare genetic rare disorders that manifest as blistering or erosion of the skin. In some cases, the epithelial lining of other organs can be affected.  There are 4 types of genetically inherited epidermolysis bullosa and in order of prevalence they are; Simplex (EBS), Dystrophic (DEB), Junctional (JEB) and Kindler. In the more severe forms of the disease, blistering can lead to deformities including fusion of the fingers and toes, secondary skin infections, sepsis and even death. Children with this condition are often in constant pain and limited to their ability to play/interact with other children due to the fear of additional skin lesions.

To learn more about epidermolysis bullosa, visit our patient care conference coverage at http://www.raredr.com/conferences/debra2014

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