Regulatory & Policy
Spinal MRI findings trigger FDA hold, derail RegenxBio’s RGX-121 filing
The FDA's clinical hold on Navsunli (clemidsogene lanparvovec-sngl [RGX-121]), an investigational adeno-associated virus (AAV) gene therapy for...
ADARx's onvuzosiran secures FDA Fast Track for twice-yearly HAE prophylaxis approach
San Diego-based ADARx Pharmaceuticals, Inc. received US FDA Fast Track Designation for onvuzosiran (ADX-324), a prekallikrein (PKK)-targeting small...
FDA fast tracks Erasca’s pan-RAS molecular glue after early pancreatic cancer responses
San Diego-based Erasca, Inc. (Nasdaq: ERAS) announced that the US FDA has granted Fast Track Designation (FTD) to ERAS-0015, an oral pan-RAS molecular glue,...
FDA accepts United Therapeutics' ralinepag NDA for PAH
The US FDA has accepted for review a New Drug Application from United Therapeutics Corporation (Nasdaq: UTHR) for ralinepag, an investigational prostacyclin...
FDA extends deramiocel review three months as Capricor narrows indication to upper limb function
The US FDA has extended the Prescription Drug User Fee Act (PDUFA) target action date for deramiocel (CAP-1002), San Diego-based Capricor Therapeutics'...
HHS seeks input on new tiers for federal vaccine recommendations
Category: Regulatory Policy | Regulatory Body: HHS | Geography: United States | Policy Instrument: Request for Information
Orum moving CD123-targeted DAC into first-in-human testing against AML
FDA clearance of an investigational new drug application for ORM-1153, a degrader-antibody conjugate (DAC) designed to deliver targeted protein degradation...
Scholar Rock withdraws EU apitegromab filing over manufacturing site issue
Cambridge, Massachusetts-based Scholar Rock (Nasdaq: SRRK) announced a series of regulatory updates on August 24, 2026, confirming that its Biologics...
FDA sets cross-center framework for digital measures in clinical trials
Category: Clinical Trial Reform | Regulatory Body: FDA (CBER, CDER, CDRH, OCE) | Geography: United States | Instrument: Guidance document
CStone to put EGFR/HER3 bispecific ADC to first-in-human test in China
China's National Medical Products Administration (NMPA) has cleared CStone Pharmaceuticals (HKEX: 2616) to begin clinical trials of CS5007, an EGFR/HER3...
China's CDE launches PIONEER Program for cell and gene therapy products
Category: Regulatory Policy | Regulatory Body: CDE (Center for Drug Evaluation, NMPA) | Geography: China | Instrument: Initiative launch
FDA grants Fast Track Designation to Rein's LTI-03 for idiopathic pulmonary fibrosis
Austin, Texas-based Rein Therapeutics (Nasdaq: RNTX) announced that the US FDA has granted Fast Track Designation to LTI-03, a first-in-class inhaled...
FDA grants Nuvation fast-track status for safusidenib in IDH1-mutant glioma
New York-based Nuvation Bio Inc. (NYSE: NUVB) announced that the US FDA has granted Fast Track Designation to safusidenib, an oral, brain-penetrant,...
Hengrui's licancopan wins China PNH approval after outperforming eculizumab in Phase III
China's National Medical Products Administration (NMPA) has approved Licancopan fumarate (HRS-5965) capsules, developed by Chengdu Suncadia Medicine Co.,...
FDA sets out potency testing approach for personalized cancer vaccines
Category: Regulatory Policy | Regulatory Body: FDA/CBER | Geography: United States | Policy Instrument: Guidance document | Topics: Potency assay...
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