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 Rastogi4 days ago

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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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Ocumension Therapeutics Reports NMPA NDA Acceptance of OT-301 to Lower Intraocular Pressure

Shots: The NMPA has accepted NDA for OT-301(bimatoprost grenod; NCX 470) to lower intraocular pressure in patients with open-angle glaucoma or ocular hypertension The NDA was backed by the P-III (Mont Blanc & Denali) trial data, where Mont Blanc’s 1EP showed robust efficacy & safety, plus Denali met its 1EP of non-inferiority of OT-301 as…

ByRidhi RastogiSeptember 11, 2026

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Pharvaris Reports the P-III (CHAPTER-3) Trial Data on Deucrictibant XR for Hereditary Angioedema (HAE)

Shots: Pharvaris has reported the P-III (CHAPTER-3) trial (N=85) assessing deucrictibant XR tablet (40mg, QD, PO; n=55) vs PBO (n=30) for the prevention of attacks in adolescents & adults with HAE Trial met all 1 & 2EPs, reducing monthly mean HAE attack rates by 83% overall & 87% in Type 1 or 2 HAE (n=80),…

ByRidhi RastogiSeptember 9, 2026

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Belite Bio Submits NDA to Japan’s MHLW for Tinlarebant in Stargardt Disease Type 1

Shots: Belite Bio submitted a New Drug Application (NDA) to Japan’s MHLW for once-daily oral tinlarebant (LBS-008) for the treatment of Stargardt disease type 1 (STGD1) The NDA was submitted under Japan’s Sakigake Designation System, following tinlarebant’s designation as a Sakigake pharmaceutical product; if approved, it could become the first approved treatment for STGD1 and…

ByPrince GiriSeptember 8, 2026

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Ionis’ Zanvastro (Zilganersen) Receives US FDA Approval for Alexander Disease (AxD)

Shots: FDA has approved Zanvastro (50mg, Q3M, intrathecal injection) for the treatment of AxD in pediatric & adult pts, with launch expected in the coming wks. FDA also granted Ionis a Rare Pediatric Disease PRV Approval was backed by the Phase I-III trial assessing Zanvastro (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for…

ByRidhi RastogiSeptember 4, 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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Amylyx Pharmaceuticals Reports Topline P-III (LUCIDITY) Trial Data on Avexitide in Post-Bariatric Hypoglycemia

Shots: The P-III (LUCIDITY) trial assessed avexitide (90mg, SC, QD) vs PBO in 78 adults with Post-Bariatric Hypoglycemia (PBH) following Roux-en-Y gastric bypass (RYGB) surgery Trial met its 1EP, showing a 55% reduction in the composite rate of Level 2 & Level 3 hypoglycemic events through Wk. 16. All 2EPs were also met, with consistent, highly…

ByRidhi RastogiAugust 18, 2026

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UroGen Submits NDA for UGN-103 to the US FDA for Recurrent LG-IR-NMIBC

Shots: The US FDA has received an NDA for UGN-103 (mitomycin; intravesical) to treat adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) The NDA is supported by the P-III (UTOPIA) trial in adults with recurrent LG-IR-NMIBC, where UGN-103 achieved a 77.8% 3mos. CR rate & a 94.5% 6mos. DoR by Kaplan-Meier estimate, consistent…

ByRidhi RastogiAugust 18, 2026

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

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

Ocumension Therapeutics Reports NMPA NDA Acceptance of OT-301 to Lower Intraocular Pressure

Pharvaris Reports the P-III (CHAPTER-3) Trial Data on Deucrictibant XR for Hereditary Angioedema (HAE)

Belite Bio Submits NDA to Japan’s MHLW for Tinlarebant in Stargardt Disease Type 1

Ionis’ Zanvastro (Zilganersen) Receives US FDA Approval for Alexander Disease (AxD)

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

Amylyx Pharmaceuticals Reports Topline P-III (LUCIDITY) Trial Data on Avexitide in Post-Bariatric Hypoglycemia

UroGen Submits NDA for UGN-103 to the US FDA for Recurrent LG-IR-NMIBC

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