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Home » Rare disease therapies from Sanofi, Eiger and PTC backed by Europe’s CHMP
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Rare disease therapies from Sanofi, Eiger and PTC backed by Europe’s CHMP

May 20, 2022
By Richard Staines
The first therapies for several rare diseases were among medicines given the green light by European regulators at their monthly meeting. The EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for Sanofi SA’s Xenpozyme (olipudase alfa) for two types of Niemann-Pick disease and Eiger Biopharmaceuticals Inc.’s Zokinvy (lonafarnib) for children with Hutchinson-Gilford progeria syndrome or progeroid laminopathies. PTC Therapeutics Inc.’s Upstaza (eladocagene exuparvovec), the first medicine for adults and children with aromatic L-amino decarboxylase deficiency, was also backed by the CHMP.
BioWorld Regulatory Rare disease Europe CHMP EMA

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