The US FDA Accepts Ultragenyx’s Resubmitted BLA for UX111 in Sanfilippo Syndrome Type A

Shots: The US FDA has accepted the resubmitted BLA of UX111 (rebisufligene etisparvovec) for accelerated approval in Sanfilippo syndrome Type A (MPS IIIA), with a PDUFA action date of Sep 19, 2026 UX111 BLA is supported by up to 8yrs. of follow-up data showing durable clinical benefit & acceptable safety. Also, during prior late-cycle review,…

ByRidhi Rastogi5 days ago

NEWS

Regeneron Wins US FDA Approval for Eylea HD with Up to 5-Month Dosing in wAMD and DME

Shots: FDA has approved extending Eylea HD dosing intervals up to 20wks. for wet age-related macular degeneration (wAMD) & diabetic macular edema (DME) after 1yr. of response based on visual & anatomic outcomes As a part of approval, the Eylea HD label will incl. 96wk. PULSAR (wAMD; n=583) & PHOTON (DME; n=395) data, where 71%…

ByRidhi Rastogi5 days ago

NEWS

The US FDA Grants Accelerated Approval Denali Therapeutics’ Avlayah (Tividenofusp alfa-eknm) for Hunter Syndrome (MPS II)

Shots: FDA has granted accelerated approval to Avlayah, an enzyme replacement therapy (ERT), for the treatment of presymptomatic or symptomatic MPS II pediatric pts (≥5kg), along with a RPD PRV to Denali; US availability is expected shortly Approval was based on the P-I/II trial of Avlayah (QW) in 47 pts, incl. ERT-naïve (n=15) & previously…

ByRidhi RastogiMarch 26, 2026

NEWS

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

Shots: The US FDA has accepted the NDA of bezuclastinib for the treatment of NonAdvSM, with no advisory committee meeting planned or review issues identified yet (PDUFA: Dec 30, 2026) NDA was backed by SUMMIT trial, which met its 1 & 2EPs, showing sustained, deepening clinical benefit through 48wks., plus impact on bone mineral density…

ByRidhi RastogiMarch 18, 2026

NEWS

AstraZeneca and Daiichi Sankyo’s Enhertu Receives US FDA Priority Review for HER2+ Early Breast Cancer

Shots: FDA has granted priority review to Enhertu’s sBLA for adults with HER2+ breast cancer who have residual invasive disease after neoadjuvant HER2 targeted treatment, with submission to be reviewed under Project Orbis (PDUFA: Q3’26); review is also ongoing in the EU & Japan sBLA was backed by the global P-III (DESTINY-Breast05) trial assessing Enhertu…

ByRidhi RastogiMarch 9, 2026

NEWS

Merck and Eisai Present P-III (LITESPARK-011) Trial Data on Welireg + Lenvima in Advanced Renal Cell Carcinoma (RCC) at ASCO GU 2026

Shots: Merck & Eisai has reported the P-III (LITESPARK-011) trial data assessing Welireg (belzutifan; 120mg, PO, QD) + Lenvima (lenvatinib; 20mg, PO, QD) vs cabozantinib in 747 pts with advanced RCC whose disease progressed on or after anti-PD-1/ PD-L1 therapy At mFU of 29mos., trial met its 1EP of improved PFS by 30% (mPFS: 14.8 vs…

ByRidhi RastogiMarch 3, 2026

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Merck Presents P-III (LITESPARK-022) Trial Data on Keytruda + Welireg in Clear Cell Renal Cell Carcinoma (RCC) at ASCO GU 2026

Shots: Merck has reported the P-III (LITESPARK-022) trial data assessing Keytruda (400mg, Q6W, IV) + Welireg (120mg, QD, PO) vs Keytruda + PBO for 1yr. in 1,841 pts with clear cell RCC following nephrectomy At 28.4mos. mFU, the trial showed improved DFS (1EP), reducing the risk of disease recurrence or death by 28%, with mDFS…

ByRidhi RastogiMarch 2, 2026

NEWS

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

Shots: The US FDA has accepted sNDA & granted priority review to olezarsen for the treatment of severe hypertriglyceridemia (sHTG), with a PDUFA action date of Jun 30, 2026 sNDA was backed by the P-III (CORE: n=617 & CORE2: n=446) trials assessing olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in sHTG adults…

ByRidhi RastogiFebruary 27, 2026

NEWS

Gilead Sciences to Acquire Arcellx for $7.8B

Shots: Gilead Sciences has entered into a definitive agreement to acquire Arcellx, incl. anito-cel from their 2022 collaboration & Arcellx’s D-Domain CAR technology platform, for the implied equity value of $7.8B As per the deal, Gilead, which owns ~11.5% of Arcellx, will launch a tender offer for $115/share, plus a non-transferable CVR of $5/CVR tied…

ByRidhi RastogiFebruary 23, 2026

NEWS

BMS Reports the US FDA’s NDA Acceptance for Iberdomide Combination in R/R Multiple Myeloma

Shots: The US FDA has accepted the NDA of iberdomide + daratumumab & dexamethasone (IberDd) for the treatment of pts with r/r multiple myeloma, with submission to be reviewed under Project Orbis (PDUFA: Aug 17, 2026) NDA was supported by the planned analysis of MRD negativity rates in the P-III (EXCALIBER-RRMM) trial assessing IberDd vs…

ByRidhi RastogiFebruary 18, 2026

The US FDA Accepts Ultragenyx’s Resubmitted BLA for UX111 in Sanfilippo Syndrome Type A

Regeneron Wins US FDA Approval for Eylea HD with Up to 5-Month Dosing in wAMD and DME

The US FDA Grants Accelerated Approval Denali Therapeutics’ Avlayah (Tividenofusp alfa-eknm) for Hunter Syndrome (MPS II)

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

AstraZeneca and Daiichi Sankyo’s Enhertu Receives US FDA Priority Review for HER2+ Early Breast Cancer

Merck and Eisai Present P-III (LITESPARK-011) Trial Data on Welireg + Lenvima in Advanced Renal Cell Carcinoma (RCC) at ASCO GU 2026

Merck Presents P-III (LITESPARK-022) Trial Data on Keytruda + Welireg in Clear Cell Renal Cell Carcinoma (RCC) at ASCO GU 2026

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

Gilead Sciences to Acquire Arcellx for $7.8B

BMS Reports the US FDA’s NDA Acceptance for Iberdomide Combination in R/R Multiple Myeloma

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