BridgeBio Pharma Gets FDA Regenerative Medicine Advanced Therapy Designation

By Sabela Ojea

BridgeBio Pharma said the Food and Drug Administration has granted regenerative medicine advanced therapy designation for its Canavan disease treatment BBP-812.

The commercial-stage biopharmaceutical company focused on genetic diseases on Tuesday said the FDA resignation came after it reviewed clinical data from its CANaspire Phase 1/2 clini0cal trial.

Regenerative medicine advanced therapy designations provide a pathway that can expedite the approval process of regenerative-medicine products.

The treatment has already been granted orphan drug, rare pediatric disease, and fast track designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency.

The Canavan disease is a rare neurodegenerative disease that prevents most children from meeting basic developmental milestones, such as crawling, walking, speaking, and even holding their heads up. It is a terminal diagnosis with no approved treatment to date, the company said.

Write to Sabela Ojea at sabela.ojea@wsj.com; @sabelaojeaguix

(END) Dow Jones Newswires

September 10, 2024 17:35 ET (21:35 GMT)

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