WebMD1d
First Brain-Injected Gene Therapy Approved by FDA
The FDA approval was based on the therapy’s safety and effectiveness as shown in an ongoing clinical trial involving 13 children diagnosed with AADC deficiency. According to PTC Therapeutics, the ...
MedPage Today7d
FDA Rejects Full Approval of Liver Disease Drug
On whether the available data supported a clinical benefit, 13 of the 14 panelists said no. Intercept ... information," ...
FiercePharma6d
FDA rejects Intercept's ask for Ocaliva full nod in rare liver disease, but accelerated approval remains
In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...
FiercePharma10h
Syndax ⁠scores FDA approval for Revuforj after it disappointed in another acute leukemia indication
The U.S. patient population for Revuforj in its approved indication is roughly 2,000 according to Syndax. KMT2A leukemias ...
Reuters5d
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
Nov 13 (Reuters) - The U.S. Food and Drug Administration approved PTC Therapeutics' (PTCT.O), opens new ... The drugmaker ...
FDA announces common over-the-counter cough and cold medicine doesn’t work
The Food and Drug Administration announced Wednesday its intention to remove oral phenylephrine, a common ingredient in over-the-counter cough and cold medicines, from the market.
Early FDA Approval of Revuforj Boosts Syndax Pharmaceuticals’ Prospects and Supports Overweight Rating
J.P. Morgan analyst Anupam Rama has maintained their bullish stance on SNDX stock, giving a Buy rating on November 13. Anupam Rama’s ...
Targeted Oncology3d
FDA Approves Revumenib in KMT2A-Rearranged Acute Leukemia
Revumenib is now an FDA-approved treatment for adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute ...
FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients ...
UroGen Pharma: Choice Based On Near-Term High-Value PDUFA
Urogen's UGN-102 presents a $5B market opportunity for bladder cancer treatment, despite risks. Read more about URGN stock ...
Monthly Prescribing Reference4d
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...