There are countless nuggets of healthcare precious metal buried within the almost 1000-page 21st Century Cures Act, agreed upon into law with bipartisan support at the end of 2016.

Among the riches is the Regenerative Medicine Advanced Treatment (RMAT) program, a type of Breakthrough Therapy Designation (BTD) meant for companies working with cell therapies, therapeutic tissues and the like.

North Carolina-based Humacyte claimed the first  RMAT designation on March 20, 2017, for its investigational human being acellular vessel (HAV). On Monday, the less than one-year-old rare disease company Enzyvant claimed the second  along with a BTD to boot.

The compound at hand will be RVT-802, a novel biologic therapy Enzyvant licensed through Duke University  in a deal announced earlier this  season. The initial target is DiGeorge syndrome (DGS), a rare hereditary disease that presents with a host of potentially fatal problems such as heart and immune system defects, a cleft taste buds, low calcium levels in the blood, and delayed advancement.

Enzyvant expects to file a biologics permit application (BLA) in early 2018 for a subset of individuals – around one percent –   who have what exactly is known as complete DiGeorge Syndrome (cDGS).   That equates to a market of just 10-20 children each year in the United States, though the number of indications will ideally expand stated CEO  Alvin Shih via email.

“ We’ re doing additional research to understand if additional potential applications for RVT-802, but our first focus on is cDGS, where we think the clinical benefit is usually most clearly demonstrated to date, ” Shih said.

Children born with cDGS have a severe insufficient thymus tissue, a necessary component of the immune system. With a drastically destabilized immune system, they typically succumb to an infection before age group two.

Enzyvant’ s RMAT-designated therapy is constructed around a proprietary process for harvesting, culturing, and using allogeneic (donor) thymic tissue. While it can’ t deal with the underlying defect in chromosome 22, RVT-802 is designed to recover some of the missing immune function. According to a company statement, initial clinical data show a survival rate of more than 70 percent for those treated with the therapy.

“ The existing data were sufficient as preliminary clinical proof that RVT-802 may address the medical unmet require in complete DiGeorge syndrome, which is a requirement for both BTD and RMAT, ” Shih said.

Plenty of that data came through the work of Mary Louise Markert, a professor of pediatrics at Duke that has treated over 70 patients and amassed decades associated with experience in the field.

Along with addressing an unmet need, the FDA outlines two other basic needs to be considered for RMAT designation: The product must be in neuro-scientific regenerative medicine and it must intend to treat, modify, invert, or cure a serious or life-threatening disease or situation.

There are substantial rewards for those that are profitable. Sponsors of the product become eligible for earlier and more included interactions with FDA to streamline the development plus review process, as happens with other breakthrough therapy designations. RMAT-designated therapies could also qualify for priority review and more rapid marketing approval and a wider scope of evidence might be submitted. That includes real-world clinical evidence, such as patient registries, electronic health records, qualitative data from clinical studies and post-approval monitoring of patients treated with the therapy.

“ The RMAT designation is so new, it’ s hard to know exactly what to expect, ” Shih mentioned. “ We know the RMAT designation confers increased relationships with the agency, as well as access to expedited review pathways with the FDA, all in an effort to expedite product development. We look forward to dealing with the Agency to make this therapy available to patients who require it. ”

A subsidiary of Vivek Ramaswamy’ s  Roivant Sciences group, Enzyvant was shaped in July 2016, with support from Plexcera Therapeutics. RVT-802 is one of two drugs in the pipeline, another is a recombinant human acid ceramidase (rhAC therapy) meant for Farber disease.

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