Analysts expect strong uptake for the antisense oligonucleotide (ASO) given the high unmet need in SMA and positive data in multiple studies, while the first ASO approved this year – Sarepta’s Exondys 51 – has seen some pushback from payers despite the lack of DMD therapies.
Biogen and partner Ionis Pharmaceuticals Inc. are ending 2016 with US FDA approval for Spinraza (nusinersen) in the treatment of spinal muscular atrophy (SMA), marking the second antisense drug approved this year to treat a rare neurodegenerative disease.
Spinraza follows in the footsteps of the controversial approval of Sarepta Therapeutics Inc.’s Exondys 51 (eteplirsen) for Duchenne muscular...
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The Swiss pharma company is rebalancing the portfolio, emphasizing best-in-class drugs and moving into new areas like cardiovascular and metabolic disease.
In this week's episode: Trump’s UK drug pricing win; Novo explains semaglutide Alzheimer's rationale; J&J and Novartis on this year’s biggest M&A deals; a look at the mixed performance of CAR-Ts; and Akeso’s goals beyond ivonescimab.
Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.