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Catalyst Pharma’s LEMS drug Firdapse wins FDA nod, as regulators keep the approval engine running at record speed

Turns out, a web error by a company contractor that tipped off investors Catalyst Pharmaceuticals’ $CPRX Firdapse had won approval earlier in the week offered alert traders a quick score. The formal notice that the FDA green-lighted the treatment for the rare neuromuscular disease came through Wednesday evening.

The drug is to be used in patients with Lambert-Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disease that affects about 1 in every 300,000 people. In patients with LEMS, the body’s immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts the ability of nerve cells to send signals to muscle cells, causing weakness and fatigue that typically culminates in difficulties with daily functioning. LEMS is often associated with other autoimmune diseases, and commonly occurs in patients with small cell lung cancer.

Until Firdapse, there was no specific FDA-approved treatment for LEMS, the agency said. Catalyst expects to make the drug commercially available early in the first quarter of 2019.

On Tuesday, the company issued a statement saying it had not yet received word from the FDA on the status of its marketing application, even though “a contractor working on a web page unintentionally allowed a live screen to be seen that reflected that the possible approval of the company’s NDA had already been received.”

Edward Nash

The drug, which is sold by BioMarin $BMRN in Europe where it has been approved since 2009, was given all the bells and whistles by the FDA — breakthrough therapy status, orphan drug designation and priority review — to hasten its path toward approval. But the Coral Gables, Florida-based biotech has not had an easy time pushing for approval – the company was forced to conduct an additional trial and  shrink its workforce by about third in 2016, after the FDA deemed its marketing application incomplete.

Firdapse brought in 18.8 million in European sales for BioMarin last year. But in the United States, it is estimated to bring in $375 million in 2025 for Catalyst, SunTrust Robinson Humphrey’s Edward Nash wrote in a note.

Nash estimated the drug will carry a wholesale price of $300,000 per patient per year — in-line with other orphan drugs such as Procysbi, Gattex and Ravicti — and forecasted 5% annual price hikes. Catalyst will likely generate Firdapse sales of about $17 million next year, he added.

Firdapse is also being investigated for use in other autoimmune conditions in two late-stage trials — in patients with congenital myasthenic syndromes (CMS) and MuSK antibody positive Myasthenia Gravis (MuSK-MG), respectively. If approved in these two conditions, Nash estimates Catalyst could further rake in a combined $491 million in 2025.

The approval marks the FDA’s 55th of the year, surpassing further its record set in 1996 of 53 approvals.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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