Sage takes to the FDA fast track with its lead rare disease drug

Sage CEO Jeff Jonas

Fresh off grossing $103.5 million in an overstuffed IPO, Cambridge, MA's Sage Therapeutics ($SAGE) has secured the promise of a speedy FDA review for its top prospect, a treatment for a rare seizure disorder.

The agency has granted its fast-track designation to SAGE-547, an intravenous treatment for patients with a serious form of the life-threatening status epilepticus (SE). The seizure condition affects about 150,000 people in the U.S., according to Sage, and those who fail repeated treatments--including medically induced comas--are diagnosed with super-refractory SE, for which there are no approved therapies.

Sage's treatment works by modulating the nervous system's GABAA receptors to tamp down seizures, and the drug has shown promise in early research. Preliminary data from a Phase I/II trial show that '547 successfully weaned the first four patients off of their anesthetic agents during treatment, meeting the study's efficacy endpoint, Sage said.

The FDA's fast-track program is reserved for drugs with the potential to treat a serious condition and meet an unmet medical need, according to the agency, and treatments in the program are eligible for more frequent feedback, rolling regulatory review and accelerated approval.

For Sage, the FDA's move is a confirmation of both '547's potential and the seriousness of super-refractory SE, CEO Jeff Jonas said.

"The receipt of orphan drug designation earlier this year for status epilepticus and the fast track designation are both significant regulatory milestones for SAGE-547, and we will continue to work closely with the FDA to advance our lead compound and the additional programs in our pipeline for the treatment of life-threatening CNS disorders," Jonas said in a statement.

The good news for '547 comes on the heels of Sage's broadly successful debut on Wall Street last week, which saw the biotech's shares rise more than 60% after opening and augmented its recent $38 million round with another heap of cash.

Beyond its lead drug, Sage has the preclinical '689, designed to treat adjunctive SE, and '217, designed to be a maintenance therapy for the disease.

- read the announcement