Protocols

NICE backs Akcea’s Tegsedi; EMA restricts Lemtrada use while it probes safety concerns

UK’s NICE has endorsed the use of Akcea’s inotersen (branded as Tegsedi) to treat patients with a rare inherited condition called hereditary transthyretin-related amyloidosis (hATTR), after the Ionis $IONS sister company offered the agency an “improved commercial agreement.” This counts as a win for Akcea, which has been competing against Alnylam’s $ALNY Onpattro, although the dataset supporting its approval is seen as a runner up to Onpattro. The UK has roughly 150 hATTR patients, according to NICE.

Last week, the EMA asked doctors to limit the use of Sanofi’s $SNY Lemtrada to adults with relapsing-remitting multiple sclerosis that is highly active despite treatment with at least two disease-modifying therapies or in patients that cannot take any other treatments. The health regulator is restricting the drug’s use while it investigates reports of immune-mediated conditions and problems with the heart and blood vessels with the medicine, including fatal cases.

→ Charles River Laboratories $CRL and the CHDI Foundation have extended their long running alliance on Huntington’s disease by another 5 years, they said last week. They initially began work together back in 2005 and are now adding multidisciplinary activities to the pact.


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

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