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FDA bestows 'Breakthrough Therapy' status to drug for fatal infant disease

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Synageva BioPharma ($GEVA) has joined the growing ranks of companies with "Breakthrough Therapy" recognition from the FDA. The agency has given the coveted status to the Lexington, MA-based biotech company's experimental treatment for use against early onset lysosomal acid lipase deficiency, an inherited disorder that typically kills infants within the first 6 month of life, according to the company.

The "Breakthrough" tag puts Synageva's therapy, sebelipase alfa, on the inside track for rapid development and review for U.S. approval for early onset LAL deficiency or Wolman disease. With more clinical evidence, the company said it could also gain this designation from the FDA for a late-onset form of the lysosomal storage disorder. The company has an underway Phase III study in late-onset patients and a Phase II/III trial for the infant form of the disease.

The company's stock price was flat as of 11:58 am Eastern time today, down less than 1% to $39.58 per share.

Synageva has become one of only a few companies outside of the ranks of big pharma or biotech groups to join the breakthrough club, with Johnson & Johnson ($JNJ), AbbVie ($ABBV), Pfizer ($PFE), Novartis ($NVS) and other industry giants taking up the majority of memberships thus far. Synageva is closer in size to Genmab, which got the FDA's "Breakthrough" status for its anticancer therapy daratumumab for multiple myeloma this month. Johnson & Johnson is partnered on Genmab's drug, however.   

Lysosomal acid lipase deficiency robs patients of an enzyme that breaks down fats and leads to buildups and damage in the liver and blood vessels. There are no approved therapies for the rare genetic disease, yet Synageva aims to change that with the help of the FDA.

"We are deeply aware of the devastating impact this disease has on infants who often die within the first 6 months of life because of this disease," Anthony Quinn, Synageva's chief medical officer, stated. "Our ongoing Phase II/III trial delivers hope for these infants and their families."

- here's Synageva's release
- and an item from The Boston Globe

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