Bayer’s Hyrnuo (Sevabertinib) Gains the US FDA Accelerated Approval for 1L Treatment of Non-Squamous HER2-Mutated NSCLC

Shots: The US FDA has granted accelerated approval to Bayer’s Hyrnuo (sevabertinib) for the 1L treatment of adults with locally advanced or metastatic non-squamous NSCLC harboring HER2/ERBB2 TKD-activating mutations Approval was supported by ORR & DoR results from the Cohort F of the P-I/II (SOHO-01) trial evaluating the efficacy & safety of Hyrnuo in treatment-naïve…

ByRidhi RastogiSeptember 10, 2026

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Nanoscope Therapeutics Reports the US FDA BLA Acceptance of Mogenry for Retinitis Pigmentosa

Shots: The US FDA has accepted BLA of Mogenry (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration for pts with retinitis pigmentosa (RP) having severe vision loss BLA was supported by the P-I/IIa trial, & the P-IIb/III (RESTORE) trial, where RESTORE met its 1 & 2EPs, showing improvements in visual acuity at…

ByRidhi RastogiSeptember 9, 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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Definium Therapeutics Receives FDA Breakthrough Therapy Designation (BTD) for DT120 in MDD

Shots: Definium Therapeutics announced that the US FDA granted BTD to DT120 (lysergide) orally disintegrating tablet (ODT) for the treatment of major depressive disorder (MDD), following positive P-III results In the P-III Emerge study, a single 100 µg dose of DT120 ODT demonstrated an 8.1-point PBO-adjusted improvement in MADRS total score at wk. 6 (p<0.0001),…

ByPrince GiriSeptember 8, 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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AstraZeneca Receives the US FDA Approval for Etcamah (Camizestrant) to Treat Advanced Breast Cancer

Shots: FDA granted accelerated approval to Etcamah + a CDK4/6 inhibitor for HR+/HER2− LA/M breast cancer with an ESR1 mutation emerging during AI + CDK4/6 inhibitor therapy, alongside approval of a ctDNA-based CDx for detecting ESR1 resistance mutations Approval was based on the P-III (SERENA-6) trial assessing Etcamah + CDK4/6 inhibitor vs AI (anastrozole/letrozole) +…

ByRidhi RastogiSeptember 7, 2026

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Milestone Pharmaceuticals Reports the NMPA Approval of Etripamil Nasal Spray for PSVT

Shots: The NMPA has approved etripamil nasal spray for the treatment of paroxysmal supraventricular tachycardia (PSVT) in adults Approval was based on data from the global P-III (RAPID) trial & a Chinese P-III (JX02002) study. JX02002 met its 1EP, showing etripamil’s superiority over PBO in converting PSVT to sinus rhythm within 30min. NMPA approval triggered…

ByRidhi RastogiSeptember 4, 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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Biogen and Eisai Report NMPA Approval of Leqembi (SC) as an Initiation Dose for Early Alzheimer’s Disease

Shots: The NMPA has approved Leqembi (SC autoinjector/SC-AI) as an initiation dose administered as 500mg given QW as two consecutive 250mg injections, each delivered in ~15secs, for the treatment of early Alzheimer's disease Approval was supported by 18mos. P-III Clarity AD data & multiple SC administration sub-studies (incl. Chinese cohort), with SC-AI 500mg (QW) showing…

ByRidhi RastogiSeptember 3, 2026

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uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

Shots: The US FDA has received a BLA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130) for the treatment of Huntington’s disease, plus a request for Priority Review BLA was supported by the 3yr. data analysis from the P-I/II trial of AMT-130 vs a propensity score-matched external control derived from the Enroll-HD natural history database;…

ByRidhi RastogiSeptember 2, 2026

Bayer’s Hyrnuo (Sevabertinib) Gains the US FDA Accelerated Approval for 1L Treatment of Non-Squamous HER2-Mutated NSCLC

Nanoscope Therapeutics Reports the US FDA BLA Acceptance of Mogenry for Retinitis Pigmentosa

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

Definium Therapeutics Receives FDA Breakthrough Therapy Designation (BTD) for DT120 in MDD

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

AstraZeneca Receives the US FDA Approval for Etcamah (Camizestrant) to Treat Advanced Breast Cancer

Milestone Pharmaceuticals Reports the NMPA Approval of Etripamil Nasal Spray for PSVT

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

Biogen and Eisai Report NMPA Approval of Leqembi (SC) as an Initiation Dose for Early Alzheimer’s Disease

uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

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