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FDA Grants Breakthrough Therapy and Regenerative Advanced Therapy Designation to Enzyvant’s RVT-802

BASEL, Switzerland, April 17, 2017 /PRNewswire/ — Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted RVT-802, Enzyvant’s investigational tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), Breakthrough Therapy designation as well as Regenerative Medicine Advanced Therapy (RMAT) designation. Both designations confer special access to the Office of Tissues and Advanced Therapies for development guidance as well as expedited review pathways.

“We are delighted that RVT-802 has received Breakthrough Therapy designation, and we are honored that it is among the very first programs to receive RMAT designation as well,” said Dr. Alvin Shih, Chief Executive Officer of Enzyvant. “We look forward to working very closely with the FDA to make our therapeutic candidate, RVT-802, available to cDGS patients as swiftly and responsibly as possible.”

“Today’s announcement offers hope that in the future children with complete DiGeorge will have timely access to this potentially life-saving therapy,” stated Dr. M. Louise Markert, Professor of Pediatrics at Duke University and the inventor of RVT-802.

“On behalf of the entire Roivant family, we are proud of this critical milestone for a promising therapy,” said Vivek Ramaswamy, founder and Chief Executive Officer of Roivant Sciences. “This is an important moment for our team, and most significantly for families confronting this terrible disease. We look forward to supporting Enzyvant in its mission to deliver RVT-802 and other potentially transformational therapies to patients suffering from rare diseases.”

Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for such designation include preliminary clinical evidence that the drug may have substantial improvement on at least one clinically significant endpoint over available therapies.

RMAT designation is a component of the 21st Century Cures Act, signed into law in December 2016. Eligible drugs include cell therapies, therapeutic tissue engineering products, human cell and tissue products, or combination products using such therapies or products. To receive this designation, eligible drugs must be intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and have preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such disease or condition.

Enzyvant is the second company to announce the receipt of RMAT designation for an investigational drug and the first to announce the simultaneous receipt of RMAT and Breakthrough Therapy designations.

 

About Complete DiGeorge Syndrome

Complete DiGeorge Syndrome (cDGS) represents approximately one percent of all patients with DiGeorge Syndrome, a congenital condition. The three main characteristics of cDGS are congenital heart disease, hypoparathyroidism (leading to low calcium levels), and athymia (lack of thymus tissue). Athymia leads to severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGS is uniformly fatal if untreated, with death typically occurring in the first two years of life due to susceptibility to infection.

About RVT-802

RVT-802 is an investigational biologic therapy using proprietary processes to harvest, culture, and apply allogeneic thymic tissue for the treatment of primary immune deficiency resulting from cDGS. Preliminary clinical results suggest a survival rate of over 70% for treated patients. In addition to Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation, RVT-802 has also been granted orphan drug designation by the FDA. Enzyvant anticipates a potential BLA filing in the first half of 2018. RVT-802 is investigational and not approved for commercial use by the FDA or other health regulators at this time.

About Enzyvant

Enzyvant is a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases. Enzyvant is currently preparing a clinical trial of RVT-801, an investigational enzyme replacement therapy for the treatment of acid ceramidase deficiency (ACD), a rare lysosomal storage disease which manifests as Farber disease. Enzyvant is also advancing the development of RVT-802, an investigational biologic therapy for complete DiGeorge Syndrome. Enzyvant plans to develop treatments for additional rare diseases with high unmet need.

About Roivant Sciences

Roivant Sciences delivers R&D solutions to the biopharmaceutical industry and academic institutions through partnerships designed to realize the full potential of promising biomedical research. Roivant advances its drug pipelines through wholly- or majority-owned Vants, including Axovant (neurology), Myovant (women’s health and prostate cancer), Dermavant (dermatology), Enzyvant (rare diseases), and Urovant (urology). Roivant’s partners include Merck, Takeda Pharmaceuticals, GlaxoSmithKline, Eisai, Vertex Pharmaceuticals, Duke University, and Cincinnati Children’s Hospital Medical Center. Our long-range mission is to reduce the time and cost of developing new medicines for patients and to share those savings with the healthcare system.

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