Ramaswamy’s Enzyvant scores a double-barreled FDA commitment to help hustle a rare disease drug through its BLA

Alvin Shih

Love him or hate him — and there are plenty of people in both camps — Vivek Ramaswamy’s companies move fast.

Just a little more than three months ago Enzyvant, Ramaswamy’s rare disease biotech, in-licensed an experimental drug for extraordinarily rare cases of complete DiGeorge Syndrome from Duke University, with eyes already firmly focused on a biologics marketing application for the newly dubbed RVT-802.

Today it’s announcing that it won both the breakthrough therapy designation and the regenerative medicine advanced therapy (RMAT) designation — the first time any drug has won a dual inside track at the FDA and only the second RMAT on record. RMAT was delivered by the 21st Century Cures Act, which includes its own revved up application process as well as loosened rules on any follow-up data that might be needed post-approval.

Congress created the BTD to get the FDA to open its doors to the most promising new therapies in the pipeline.

So let’s say the FDA is doubly committed to seeing this therapy hit Enzyvant’s plans to file a BLA next year.

Bermuda-based Roivant Sciences and Plexcera Therapeutics created  Enzyvant Sciences last July, launching with a recombinant human acid ceramidase (rhAC), an enzyme replacement therapy for the treatment of patients with Farber disease.

The private Enzyvant is one of a group of companies Ramaswamy – who’s backed by some very deep pocket investors as well as a pair of big IPOs — has ramped up in the last couple of years under the Roivant banner. His flagship company is Axovant $AXON, which will be releasing Phase III data for a symptomatic Alzheimer’s drug this fall under new CEO David Hung. Myovant Sciences $MYOV is his other public biotech, focused on women’s health and prostate cancer.

“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.”

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