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 Rastogi1 day ago

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Bayer’s Kerendia Secures US FDA Approval for Chronic Kidney Disease Associated with T1D

Shots: The US FDA has approved Kerendia (finerenone, 10 & 20mg) for the treatment of adults with chronic kidney disease (CKD) associated with type 1 diabetes (T1D) Approval was supported by the global P-III (FINE-ONE) trial assessing Kerendia (10 or 20mg, QD) + SoC vs PBO in 242 pts with CKD associated with T1D Kerendia +…

ByRidhi Rastogi2 days ago

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Biogen and Eisai Report MHLW Approval of Leqembi Pen (SC) for Early Alzheimer’s Disease

Shots: The MHLW has approved Leqembi Pen (SC autoinjector) administered as 500mg given QW as two pens, each delivered in ~15secs, for the treatment of early Alzheimer's disease Approval was supported by 18mos. P-III Clarity AD data in pts with mild cognitive impairment (MCI) due to AD or mild AD dementia & multiple SC administration…

ByRidhi Rastogi3 days ago

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Lundbeck Reports the MHLW Approval of Vyepti (Eptinezumab) for the Preventive Treatment of Migraine

Shots: The Japanese MHLW has approved Vyepti (eptinezumab) for the prevention of migraine attacks in adults Approval was backed by extensive clinical data, incl. P-III (PROMISE-1, PROMISE-2 & DELIVER) studies as well as P-III (SUNRISE) trial, which assessed Vyepti in a predominantly Asian population; NDA was further supported by the SUNSET LTE trial conducted in Japanese…

ByRidhi Rastogi3 days ago

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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 Rastogi4 days ago

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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 Rastogi4 days ago

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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

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Scholar Rock Reports the US FDA Approval of Isembyld (Apitegromab-mstn) to Treat Spinal Muscular Atrophy

Shots: The US FDA has approved Isembyld for the treatment of spinal muscular atrophy (SMA) in pts (≥2yrs.) who are currently receiving a SMN2-targeted treatment Approval was backed by the P-III (SAPPHIRE), which met its 1EP of 2.2-point improvement in HFMSE with Isembyld + SMN2-targeted therapy vs PBO at 1yr. in main efficacy population (n=103);…

ByRidhi RastogiSeptember 14, 2026

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Samsung Bioepis Reports MHLW Approval of its Ustekinumab Biosimilar

Shots: The MHLW has granted marketing approval to Ustekinumab BS IV infusion 130mg 「NIPRO」, a biosimilar version of Stelara (ustekinumab), for induction therapy in mod. to sev. active Crohn’s disease when other treatments are ineffective Additionally, the MHLW approved Ustekinumab BS SC Injection 45mg Syringes 「NIPRO」in Dec 2025, which was then launched in May 2026 The biosimilar…

ByRidhi RastogiSeptember 4, 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

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

Bayer’s Kerendia Secures US FDA Approval for Chronic Kidney Disease Associated with T1D

Biogen and Eisai Report MHLW Approval of Leqembi Pen (SC) for Early Alzheimer’s Disease

Lundbeck Reports the MHLW Approval of Vyepti (Eptinezumab) for the Preventive Treatment of Migraine

Curium Receives FDA Approval for Bexlutry to Treat GEP-NETs

Telix Gains US FDA Approval for Pixclara for Brain Cancer Imaging

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

Scholar Rock Reports the US FDA Approval of Isembyld (Apitegromab-mstn) to Treat Spinal Muscular Atrophy

Samsung Bioepis Reports MHLW Approval of its Ustekinumab Biosimilar

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

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