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Regulatory Intelligence & Drug Approvals1 month agoFDA Approves Therapy to Treat Ataxia in Patients with Ataxia-Telangiectasia, a Rare Genetic Disorder
FDA has approved Aqneursa (levacetylleucine) for oral suspension to treat ataxia in adults and pediatric patients with ataxia-telangiectasia weighing at least 15 kg (about 33 pounds).
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Regulatory Intelligence & Drug Approvals1 month agoXenon shares fall following depression trial pause
The occurrence of “neuropsychiatric” adverse events in testing fueled investor doubts about a drug the FDA is already reviewing for a form of epilepsy.
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Regulatory Intelligence & Drug Approvals1 month agoEli Lilly escalates oral SERD battle with FDA approval for Inluriyo, Verzenio combo
With longer progression-free survival, Jake Van Naarden, president of Lilly Oncology, told Fierce he expects most patients to choose the combination over oral SERD monotherapy.
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Regulatory Intelligence & Drug Approvals1 month agoXenon tumbles on voluntary psychiatry hold, but analysts confident in $2B epilepsy opportunity
As Xenon Pharmaceuticals announced that azetukalner has reached the FDA for focal seizures, the company also disclosed the emergence of psychosis adverse events in psychiatric trials...
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Regulatory Intelligence & Drug Approvals1 month agoSTAT+: Roche expands its Boston footprint with new research center
A gene therapy approved for Sanfilippo syndrome, good and bad news for Xenon Pharma, and more biotech news
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Regulatory Intelligence & Drug Approvals1 month agoNew Approach Methodologies (NAMs)
The FDA is leading a transformative shift in drug safety evaluation—reducing, replacing or refining animal testing with advanced, human-relevant methods that better predict how medicines work...
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Regulatory Intelligence & Drug Approvals1 month agoNew Approach Methodologies (NAMs) Database of Use Case Examples
Explore practical examples of New Approach Methodologies (NAMs) described in publicly available FDA application review materials.
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Regulatory Intelligence & Drug Approvals1 month ago
With pioneering ADHD data, Alkermes eyes orexin agonist as potential ‘company changer’
Following Takeda’s historic approval of the first orexin agonist with its narcolepsy treatment Orzeyful, Alkermes has followed up with pioneering data that looks to take the...
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Regulatory Intelligence & Drug Approvals1 month agoUltragenyx wins FDA greenlight for first Sanfilippo therapy, priced at nearly $4M
The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene...
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Regulatory Intelligence & Drug Approvals1 month agoCHMP backs Novo’s haemophilia A drug Frehemgo
Novo has moved a step closer to EU approval of its long-acting haemophilia A therapy, Frehemgo, which could be a future blockbuster.
LncRNA SBF2-AS1 Worsens Heart Injury After Attack via miR-149-5p/PHLPP2 Axis
Stigmatizing language found in over half of primary care patient records
Research report: The Influence of Renal or Hepatic Impairment on the Pharmacokinetics of Iclepertin (BI 425809): Results from Two Phase I Open-Label, Non-randomised, Single Dose, Parallel Design Studies
Novartis buys Synnovation’s PI3Kα inhibitors for $3 billion
Novartis to pay $2B upfront to take next-gen PI3Kα inhibitor from Synnovation
