IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

Shots: The US FDA has approved Aqneursa (levacetylleucine) for the treatment of ataxia in adult & pediatric pts (≥15kg) with ataxia-telangiectasia (A-T) Approval was backed by the P-III (IB1001-303) trial assessing Aqneursa vs PBO in 73 pts (4-50yrs.) with genetically confirmed A-T, which met its 1 & key 2EPs & showed that Aqneursa has a…

ByRidhi RastogiSeptember 21, 2026

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The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Shots: The FDA has granted full approval to Fayuvi (rebisufligene etisparvovec-hopf) for pediatric pts with Sanfilippo syndrome Type A, plus a Priority Review Voucher; commercial product will be available for shipment to QTCs within 30-60 days Approval was supported by pivotal Transpher A & long-term follow-up data, where Fayuvi-treated pts from the mITT population (n=17)…

ByRidhi RastogiSeptember 18, 2026

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Aptitude Receives US FDA Clearance for Next-Generation Metrix COVID Test

Shots: The US FDA has granted 510(k) clearance to the next-generation Metrix COVID Test, with development supported by BARDA. The test is cleared for OTC and professional use in CLIA-waived settings The handheld molecular test delivers approximately 3-fold higher analytical sensitivity & 30% faster results than EUA-authorized product, targeting urgent care & other decentralized settings Aptitude…

ByRidhi RastogiSeptember 17, 2026

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Cogent Biosciences Reports US FDA’s NDA Acceptance of Bezuclastinib for Advanced Systemic Mastocytosis (AdvSM)

Shots: The US FDA has accepted an NDA for bezuclastinib in pts with AdvSM, plus set a PDUFA target action date of Jun 29, 2027, with no plans to hold an advisory committee, as well as no potential review issues identified NDA was backed by the APEX trial, which showed as of Mar 31, 2026,…

ByRidhi RastogiSeptember 16, 2026

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Curium Receives FDA Approval for Bexlutry to Treat GEP-NETs

Shots: The US FDA has approved Bexlutry (lutetium Lu 177 dotatate injection) for the treatment of adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), covering foregut, midgut, & hindgut NETs Bexlutry is a radioligand equivalent developed through the 505(b)(2) regulatory pathway, targeting Lutathera. The approval is supported by published evidence & targeted bridging data demonstrating…

ByRidhi RastogiSeptember 15, 2026

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Organon Reports the US FDA sNDA Acceptance for Miudella

Shots: The US FDA has accepted the sNDA of Miudella (hormone-free copper intrauterine system) for an extended duration of use for up to 6yrs. with the PDUFA action date expected in Q2’27 Miudella was evaluated in 3 trials across US in 1904 women (17-45yrs.), where the efficacy cohort (17-35yrs.) of the P-III trial (n=1397) showed…

ByRidhi RastogiSeptember 15, 2026

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Telix Gains US FDA Approval for Pixclara for Brain Cancer Imaging

Shots: The US FDA has approved Pixclara (floretyrosine F 18) for pts (≥1mos.) as the FET-PET imaging drug for glioma, enabling PET imaging to help differentiate recurrent or progressive glioma from treatment-related changes Pixclara comprises a small-molecule targeting agent & fluorine-18; it binds with LAT1 & LAT2 transporters after IV administration, allowing the radioisotope’s emissions…

ByRidhi RastogiSeptember 15, 2026

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Cyllene Therapeutics Secures US FDA RMAT Designation for EG110A in Neurogenic Detrusor Overactivity

Shots: The US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to EG110A to treat neurogenic detrusor overactivity (NDO) with urinary incontinence The designation was based on the initial P-Ib/IIa trial data, ongoing in the US, in pts with NDO associated with spinal cord injury, with interim results to be presented at the ICS…

ByRidhi RastogiSeptember 14, 2026

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The US FDA Grants Priority Review to Takeda’s Zasocitinib in Plaque Psoriasis

Shots: The US FDA has accepted the NDA & granted priority review to zasocitinib (TAK-279) for the treatment of adults with mod. to sev. plaque psoriasis (PsO), with the PDUFA target action date in Q1’27. MAA is under EMA’s review, with further global filings planned NDA was supported by extensive data incl. global P-III (Latitude…

ByRidhi RastogiSeptember 14, 2026

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Pharming Reports the US FDA Approval of Joenja (Leniolisib) for Activated Phosphoinositide 3-Kinase Delta Syndrome

Shots: The US FDA has approved Joenja (leniolisib; 40 & 50mg, BID, PO) for the treatment of children (4-11yrs.; ≥27kg) with activated phosphoinositide 3-kinase delta (APKD) syndrome Approval was backed by a P-III trial in APKD pediatric pts (4-11yrs.), which showed reduced lymphadenopathy & increased naïve B cells over 12wks. with a favorable safety profile…

ByRidhi RastogiSeptember 14, 2026

IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Aptitude Receives US FDA Clearance for Next-Generation Metrix COVID Test

Cogent Biosciences Reports US FDA’s NDA Acceptance of Bezuclastinib for Advanced Systemic Mastocytosis (AdvSM)

Curium Receives FDA Approval for Bexlutry to Treat GEP-NETs

Organon Reports the US FDA sNDA Acceptance for Miudella

Telix Gains US FDA Approval for Pixclara for Brain Cancer Imaging

Cyllene Therapeutics Secures US FDA RMAT Designation for EG110A in Neurogenic Detrusor Overactivity

The US FDA Grants Priority Review to Takeda’s Zasocitinib in Plaque Psoriasis

Pharming Reports the US FDA Approval of Joenja (Leniolisib) for Activated Phosphoinositide 3-Kinase Delta Syndrome

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