Travere Therapeutics Reports the US FDA’s sNDA Acceptance of Filspari for Focal Segmental Glomerulosclerosis (FSGS)

Shots:The US FDA has accepted sNDA of Filspari for the treatment of FSGS, plus FDA has planned to hold advisory committee meeting to discuss the application (PDUFA: Jan 13, 2026)sNDA was supported by P-III (DUPLEX) trial & P-II (DUET) trial assessing Filspari vs irbesartan in FSGS pts; Pts completing the DUPLEX & DUET double-blind…

ByRidhi RastogiMay 16, 2025

News

The US FDA Approves Incyte’s Zynyz to Treat Adults with Squamous Cell Anal Carcinoma (SCAC)

Shots:FDA has approved Zynyz (retifanlimab-dlwr) + Pt-based CT as 1L treatment of inoperable locally recurrent or metastatic SCAC, & as monotx. for locally recurrent or metastatic SCAC progressing on or intolerant to Pt-based CT; EMA & PMDA’s application are under reviewApproval was based on P-III (POD1UM-303/InterAACT2) trial assessing Zynyz + Pt-based CT vs…

ByRidhi RastogiMay 16, 2025

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AbbVie’s Emrelis Receives the US FDA’s Accelerated Approval for NSCLC With High c-Met Protein Overexpression

Shots:The US FDA has granted accelerated approval to Emrelis (telisotuzumab vedotin-tllv) for treatment-experienced pts with locally advanced or metastatic, c-Met overexpressing, non-squamous NSCLC Approval was backed by an ongoing P-II (LUMINOSITY) study assessing Emrelis as 2L/3L treatment of c-Met overexpressing NSCLC; showing an ORR of 35% & mDoR of 7.2mos. in pts (n=84) with…

ByRidhi RastogiMay 16, 2025

News

Merck’s Welireg Receives the US FDA’s Approval for Pheochromocytoma and Paraganglioma

Shots:The US FDA has approved Welireg for the treatment of pts (≥12yrs.) with advanced, unresectable, or metastatic pheochromocytoma & paraganglioma (PPGL)Approval was based on Cohort A1 data from P-II (LITESPARK-015) trial assessing Welireg monotx. (120mg; QD) in 72 pts with unresectable or non-curable PPGL & stable blood pressure (BP <150/90 mm Hg, <135/85 mm…

ByRidhi RastogiMay 15, 2025

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MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Shots:The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental…

ByRidhi RastogiMay 13, 2025

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Cognition Therapeutics Reports Topline P-II (MAGNIFY) Trial Data of Zervimesine for Geographic Atrophy (GA)

Shots:Cognition has reported topline P-II (MAGNIFY/COG2201) trial data assessing zervimesine (200mg, QD, PO) vs PBO in adults with GA secondary to dry AMD; trial concluded after enrolling ~100 of 246 planned subjectsIn the trial, nearly 2/3 pts completed 12mos. & 1/3 completed 18mos. of dosing, showing a 28.6% slower GA progression & ~12%,…

ByRidhi RastogiMay 9, 2025

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FDA Grants Verastem Oncology’s Avmapki + Fakzynja Accelerated Approval for KRAS-Mutated Low-Grade Serous Ovarian Cancer (LGSOC)

Shots:This accelerated approval for the combination of Avmapki (avutometinib) + Fakzynja (defactinib) was granted in advance to the planned PDUFA of Jun 30, 2025, which will be available in the US within a week as a co-pack Approval was based on P-II (RAMP 201) trial assessing Avmapki (3.2mg, twice weekly) + Fakzynja (200 mg,…

ByDipanshu DixitMay 9, 2025

News

Amgen Reports the MHRA’s Approval of Tepezza to Treat Thyroid Eye Disease

Shots:UK’s MHRA has approved Tepezza (teprotumumab) for the treatment of adult pts with mod. to sev. thyroid eye disease (TED)Approval was based on extensive clinical data incl. P-III (OPTIC) trial (n=83), P-II (TED01RV) trial (n=88), P-IV (HZNP-TEP-403) trial (n=62) & P-III (OPTIC-J) trial in Japan (n=54)OPTIC trial showed that 83% (34/41) of…

ByRidhi RastogiMay 8, 2025

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Recursion Presents Preliminary P-Ib/II (TUPELO) Trial Data of REC-4881 for Familial Adenomatous Polyposis (FAP) at DDW 2025

Shots:P-Ib/II (TUPELO) trial (enrolment ongoing) assessed REC-4881 (4 or 8mg, QD) in FAP, with P-II evaluating efficacy via upper & lower endoscopy at Wk. 0, 13 (on-treatment), & 25 (off-treatment) in pts (≥55yrs.) with APC mutationsAs of Mar 17, 2025, 4mg (n=6) showed a median 43% polyp burden decrease; 5/6 had reductions of…

ByRidhi RastogiMay 5, 2025

News

Relmada Therapeutics Presents Initial P-II Trial Data of NDV-01 for High-Grade Non-Muscle Invasive Bladder Cancer (HG-NMIBC) at AUA 2025

Shots:Relmada has reported initial P-II trial data assessing NDV-01 in 20 pts with HG-NMIBC (8 BCG-naïve, 12 BCG-unresponsive), where 2 pts had CIS & 18 had papillary disease (Ta/T1); 26 pts are enrolled, with 20 reaching 3mos. & 7 reaching 6mos. assessmentsTrial showed 85% ORR, 83.3% HGRFS in Ta/T1, & 100% CR in…

ByRidhi RastogiApril 29, 2025

Travere Therapeutics Reports the US FDA’s sNDA Acceptance of Filspari for Focal Segmental Glomerulosclerosis (FSGS)

The US FDA Approves Incyte’s Zynyz to Treat Adults with Squamous Cell Anal Carcinoma (SCAC)

AbbVie’s Emrelis Receives the US FDA’s Accelerated Approval for NSCLC With High c-Met Protein Overexpression

Merck’s Welireg Receives the US FDA’s Approval for Pheochromocytoma and Paraganglioma

MavriX Bio Reports the US FDA’s IND Clearance of MVX-220 for Angelman Syndrome

Cognition Therapeutics Reports Topline P-II (MAGNIFY) Trial Data of Zervimesine for Geographic Atrophy (GA)

FDA Grants Verastem Oncology’s Avmapki + Fakzynja Accelerated Approval for KRAS-Mutated Low-Grade Serous Ovarian Cancer (LGSOC)

Amgen Reports the MHRA’s Approval of Tepezza to Treat Thyroid Eye Disease

Recursion Presents Preliminary P-Ib/II (TUPELO) Trial Data of REC-4881 for Familial Adenomatous Polyposis (FAP) at DDW 2025

Relmada Therapeutics Presents Initial P-II Trial Data of NDV-01 for High-Grade Non-Muscle Invasive Bladder Cancer (HG-NMIBC) at AUA 2025

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