BridgeBio Pharma Reports the US FDA Priority Review of Oral Infigratinib for Children with Achondroplasia

Shots: The US FDA has accepted NDA & granted Priority Review to infigratinib (PO) for the treatment of children with achondroplasia (PDUFA: Feb 4, 2027). BridgeBio is prepared to launch infigratinib upon approval Trial met its 1EP, with infigratinib improving annualized height velocity at Wk. 52 (mean treatment difference: +2.10cm/yr.) & its key 2EP of…

ByRidhi Rastogi2 days ago

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AstraZeneca Secures US FDA Priority Review for Imfinzi + Enfortumab Vedotin for Muscle-Invasive Bladder Cancer

Shots: The US FDA has accepted sBLA and granted Priority Review to Imfinzi (durvalumab) + enfortumab vedotin (EV) for the treatment of pts with MIBC who are ineligible for or have declined cisplatin-based CT (PDUFA: Q4’26). Regulatory review is ongoing in EU & Japan sBLA was based on the P-III (VOLGA) trial, which randomized 695…

ByRidhi RastogiSeptember 28, 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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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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Genentech Secures the US FDA Priority Review for Enspryng (Satralizumab) to Treat MOGAD

Shots: FDA has granted Priority Review to the sBLA of Enspryng for the treatment of myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), with a PDUFA target action date set for Jan 10, 2027. Filing is also under EMA’s review, with a decision expected in Q3’27 sBLA was supported by the P-III (METEOROID) trial data, assessing Enspryng(60,…

ByRidhi RastogiSeptember 10, 2026

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The US FDA Accepts Nicox’s NDA for NCX 470 in Glaucoma

Shots: The FDA has accepted NDA for NCX 470 (bimatoprost grenod) to lower intraocular pressure in patients with open-angle glaucoma or ocular hypertension (PDUFA: Apr 30, 2027) The NDA, submitted by US licensee Kowa, is supported by P-III (Mont Blanc & Denali) trial data showing IOP reductions of up to 10mmHg with a favorable safety…

ByRidhi RastogiAugust 28, 2026

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United Therapeutics Reports the US FDA NDA Acceptance for Ralinepag to Treat Pulmonary Arterial Hypertension

Shots: The US FDA has accepted the NDA for ralinepag to treat pulmonary arterial hypertension, with PDUFA target action date of Jun 24, 2027 NDA was supported by the P-III (ADVANCE OUTCOMES) trial assessing ralinepag vs PBO in 687 patients with PAH, where pts who completed the trial had the option to enroll in ongoing…

ByRidhi RastogiAugust 25, 2026

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GSK’s Jemperli (Dostarlimab) Secures US FDA Priority Review for dMMR/MSI-H Locally Advanced Rectal Cancer

Shots: The US FDA has accepted the sBLA under Project Orbis & granted priority review to Jemperli in previously untreated stage II/III dMMR/MSI-H locally advanced rectal cancer (PDUFA: Feb 2027), with eligibility for an earlier decision through the NPV program sBLA was based on the global P-II (AZUR-1) trial assessing Jemperli (dostarlimab) monotx. (500mg, IV, Q3W…

ByRidhi RastogiAugust 25, 2026

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BMS Reports the US FDA Accelerated Approval for Zenbexus (Iberdomide) Combination in R/R Multiple Myeloma (MM)

Shots: FDA has granted accelerated approval to Zenbexus (iberdomide) + daratumumab & hyaluronidase-fihj + dexamethasone (ZDd) for the treatment of pts with MM who received ≥1L of prior therapy incl. a proteasome inhibitor & an immunomodulatory agent Approval was supported by the P-III (EXCALIBER-RRMM) trial assessing ZDd (n=207) vs daratumumab + bortezomib + dexamethasone (DVd;…

ByRidhi RastogiAugust 14, 2026

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Belite Bio Receives the US FDA Priority Review for Tinlarebant to Treat Stargardt Disease Type 1

Shots: The US FDA has accepted & granted Priority Review to the NDA for tinlarebant for the treatment of Stargardt Disease Type 1 (STGD1), with a PDUFA target date of Feb 12, 2027 NDA was supported by the P-III (DRAGON) trial assessing tinlarebant vs PBO in STGD1 pts, showing 35.7% reduction in atrophic retinal lesion growth…

ByRidhi RastogiAugust 12, 2026

BridgeBio Pharma Reports the US FDA Priority Review of Oral Infigratinib for Children with Achondroplasia

AstraZeneca Secures US FDA Priority Review for Imfinzi + Enfortumab Vedotin for Muscle-Invasive Bladder Cancer

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

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

Genentech Secures the US FDA Priority Review for Enspryng (Satralizumab) to Treat MOGAD

The US FDA Accepts Nicox’s NDA for NCX 470 in Glaucoma

United Therapeutics Reports the US FDA NDA Acceptance for Ralinepag to Treat Pulmonary Arterial Hypertension

GSK’s Jemperli (Dostarlimab) Secures US FDA Priority Review for dMMR/MSI-H Locally Advanced Rectal Cancer

BMS Reports the US FDA Accelerated Approval for Zenbexus (Iberdomide) Combination in R/R Multiple Myeloma (MM)

Belite Bio Receives the US FDA Priority Review for Tinlarebant to Treat Stargardt Disease Type 1

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