US FDA Accepts sBLA for Merck’s Enflonsia to Expand RSV LRTD Prevention Indication

Shots: FDA has accepted the sBLA of Enflonsia to prevent RSV lower respiratory tract disease (LRTD) in children (<2yrs.) at increased risk for severe RSV disease through their second RSV season (PDUFA: Mar 22, 2027); application is also under EMA review Submissions were supported by P-III (SMART/MK-1654-007) trial assessing Enflonsia in infants & children over…

ByRidhi Rastogi3 days ago

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BridgeBio Pharma Reports the US FDA’s NDA Acceptance for Encaleret to Treat Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Shots: The US FDA has accepted NDA for encaleret to treat ADH1, with a PDUFA date of May 8, 2027, & no advisory committee planned. BridgeBio is prepared for a potential launch, while an EU MAA filing is expected in H2’26 Encaleret was evaluated in the P-III (CALIBRATE) trial, which met its 1 & 2EPs,…

ByRidhi RastogiJuly 22, 2026

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BioMarin Reports the US FDA’s sNDA Acceptance for Full Approval of Voxzogo in Achondroplasia

Shots: The US FDA has accepted an sNDA for Voxzogo (vosoritide) for full approval in children with achondroplasia (PDUFA: Feb. 28, 2027) The sNDA was supported by long-term safety & efficacy data from the ongoing 111-205, 111-208, & 111-302 studies, demonstrating clinically meaningful improvements in growth & key skeletal measures, incl. body proportionality & arm span…

ByRidhi RastogiJuly 14, 2026

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Immunome Reports the US FDA’s NDA Acceptance of Varegacestat to Treat Desmoid Tumors in Adults

Shots: The US FDA has accepted the NDA for Immunome’s varegacestat for the treatment of adults with desmoid tumors, with a PDUFA target action date of Apr 28, 2027 NDA is supported by the global P-III (RINGSIDE) trial assessing varegacestat (1.2mg, QD) vs PBO until disease progression or death in 156 pts with progressing desmoid…

ByRidhi RastogiJuly 8, 2026

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Agios Pharmaceuticals Receives US FDA Priority Review for Mitapivat to Treat Sickle Cell Disease

Shots: The US FDA has accepted the sNDA & granted priority review to mitapivat, under FDA’s accelerated approval pathway, for the treatment of Sickle Cell Disease (PDUFA: Nov 1, 2026) sNDA was supported by the global P-II/III (RISE UP) trial assessing mitapivat (100mg, PO, BID; n=138) vs PBO (n=69) in 207 pts with SCD (≥16yrs.)…

ByRidhi RastogiJuly 7, 2026

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Pharvaris Reports the US FDA’s NDA Acceptance of Deucrictibant IR for On-Demand Treatment of Hereditary Angioedema Attacks

Shots: The US FDA has accepted NDA for deucrictibant immediate-release (IR) capsule (20 mg) for the on-demand treatment of Hereditary Angioedema (HAE) attacks (PDUFA: Apr 23, 2027) NDA was backed by data from >1,300 HAE attacks. In P-III (RAPIDe-3) trial, deucrictibant IR met the 1EP & all eleven 2EPs, with median times to symptom relief,…

ByRidhi RastogiJuly 6, 2026

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Ionis Inks ex-US Deal with Recordati for Zilganersen in Alexander Disease

Shots: Ionis has granted Recordati exclusive rights to develop & commercialize zilganersen for Alexander disease outside the US, while Ionis retains US commercialization rights & will continue to lead global development Ionis will receive $30M upfront & additional milestone payments, with tiered royalties of up to the mid-20% range on annual net sales. Recordati will…

ByRidhi RastogiJune 25, 2026

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Otsuka Reports P-IIIb Trial Data on Centanafadine in ADHD and Comorbid Anxiety

Shots: Otsuka has reported topline P-IIIb trial results evaluating centanafadine XR (280mg, QD) vs PBO in 315 adults (18-65yrs.) with attention-deficit/hyperactivity disorder (ADHD) & comorbid generalized anxiety disorder and/or social anxiety disorder The trial met its 1EP, with AISRS total score improvement at Wk. 8 (-18.5 vs -12.6), evident from Wk. 1 & sustained throughout the…

ByRidhi RastogiJune 25, 2026

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Inhibrx Biosciences Reports the US FDA’s BLA Acceptance of Ozekibart to Treat Conventional Chondrosarcoma

Shots: The US FDA has accepted BLA of ozekibart (INBRX-109) for the treatment of patients with unresectable or metastatic conventional chondrosarcoma, with no filing review issues identified yet (PDUFA: Apr 14, 2027) BLA was backed by the ChonDRAgon study assessing ozekibart vs PBO in pts with metastatic or unresectable conventional chondrosarcoma, which met its 1EP…

ByRidhi RastogiJune 17, 2026

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AstraZeneca Reports FDA’s sBLA Acceptance Under Priority Review for Ultomiristo Treat IgA Nephropathy

Shots: The US FDA has accepted sBLA & granted priority review to AZ's Ultomiris (ravulizumab) for the treatment of adults with immunoglobulin A nephropathy (IgAN), with a PDUFA action date anticipated in Q4’26 sBLA was supported by the interim analysis data from the global P-III (I CAN/ALXN1210-IgAN-320) trial assessing Ultomiris (IV) vs PBO in ~510 adults with…

ByRidhi RastogiJune 15, 2026

US FDA Accepts sBLA for Merck’s Enflonsia to Expand RSV LRTD Prevention Indication

BridgeBio Pharma Reports the US FDA’s NDA Acceptance for Encaleret to Treat Autosomal Dominant Hypocalcemia Type 1 (ADH1)

BioMarin Reports the US FDA’s sNDA Acceptance for Full Approval of Voxzogo in Achondroplasia

Immunome Reports the US FDA’s NDA Acceptance of Varegacestat to Treat Desmoid Tumors in Adults

Agios Pharmaceuticals Receives US FDA Priority Review for Mitapivat to Treat Sickle Cell Disease

Pharvaris Reports the US FDA’s NDA Acceptance of Deucrictibant IR for On-Demand Treatment of Hereditary Angioedema Attacks

Ionis Inks ex-US Deal with Recordati for Zilganersen in Alexander Disease

Otsuka Reports P-IIIb Trial Data on Centanafadine in ADHD and Comorbid Anxiety

Inhibrx Biosciences Reports the US FDA’s BLA Acceptance of Ozekibart to Treat Conventional Chondrosarcoma

AstraZeneca Reports FDA’s sBLA Acceptance Under Priority Review for Ultomiristo Treat IgA Nephropathy

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