In May, the U.S. Food and Drug Administration (FDA) approved Novartis’ gene therapy Zolgensma as a one-time treatment for spinal muscular atrophy. On Monday though, the regulatory agency revealed that data manipulation was involved in the preclinical process but suggests…
Canada Urges Industry to Prepare for U.S. Drug Import Changes
Last week, the Trump administration announced it was evaluating proposals to allow consumers to buy drugs legally from Canada. HHS Secretary Alex Azar said that the Trump administration will consider new rules that would allow states, pharmacies and other parties…
Daiichi Sankyo’s Turalio Approved for Rare, Non-Cancerous Tumor
The U.S. Food and Drug Administration (FDA) approved Daiichi Sankyo’s Turalio (pexidartinib) for adults with symptomatic tenosynovial giant cell tumor (TGCT). TGCT is a rare, non-malignant tumor that can be aggressive locally. It affects the synovium-lined joints, bursae and tendon…
Sosei Heptares and Takeda Ink $1.2 Billion+ Deal to Develop GPCR Targets
Sosei Group Corporation inked a strategic multi-target partnership with Takeda Pharmaceutical to find, develop and commercialize small molecules and biologics that modulate G protein-coupled receptor (GPCR) targets. The initial focus will be on gastrointestinal targets. Under the terms of the…
Amicus Therapeutics’ Gene Therapy for Rare, Fatal Childhood Disease Shows Promise in Early Clinical Trial
Amicus Therapeutics, based in Cranbury, New Jersey, announced positive interim results from its CLN6 Batten disease gene therapy program. The Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital is running the ongoing Phase I/II clinical trial of a one-time…
