Regeneron is digging into the rare disease business
Regeneron Pharmaceuticals is one step closer to winning FDA approval for evinacumab, an intravenous treatment for a rare genetic disease that causes dangerously high cholesterol levels. If everything goes to plan, the drug could mark an evolution for a company that has made its name — and billions of dollars — treating more widespread conditions.
The FDA has set a decision date for Feb. 11, and evinacumab’s supporting data suggest the drug will win approval. But commercialization will be interesting. The drug’s indication, homozygous familial hypercholesterolemia, affects only about 1,300 people in the U.S. And, because there are other cholesterol-lowering options out there, evinacumab may be reserved for only the most severe patients.
Regeneron’s first rare-disease treatment, Arcalyst, is a commercial nonentity. Evinacumab offers a chance to build a more sustainable business in the rare-disease space, as does the company’s next drug, a treatment for the rare fibrodysplasia ossificans progressiva that is expected to go before the FDA next year.
The FDA has set a decision date for Feb. 11, and evinacumab’s supporting data suggest the drug will win approval. But commercialization will be interesting. The drug’s indication, homozygous familial hypercholesterolemia, affects only about 1,300 people in the U.S. And, because there are other cholesterol-lowering options out there, evinacumab may be reserved for only the most severe patients.
Regeneron’s first rare-disease treatment, Arcalyst, is a commercial nonentity. Evinacumab offers a chance to build a more sustainable business in the rare-disease space, as does the company’s next drug, a treatment for the rare fibrodysplasia ossificans progressiva that is expected to go before the FDA next year.
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