Pfizer’s Vyndaqel Success Interfering With Alnylam's Plans
Blockbuster Status In Sight For Pfizer Drug
Alnylam wants to emulate success of Eylea and Revlimid in amyloidosis– but Pfizer could spoil the plan.
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The first use of CRISPR/Cas9 in humans shows promising safety and efficacy against rare disease ATTR, and opens up the possibility of treating many genetic diseases.
The results are in for hATTR amyloidosis with polyneuropathy, while results for hereditary and wild-type ATTR with cardiomyopathy are expected later this year.
Givosiran was approved by the US FDA for the treatment of acute hepatic porphyria, a rare generic blood disorder, nearly three months ahead of the action date.