IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

Shots: The US FDA has approved Aqneursa (levacetylleucine) for the treatment of ataxia in adult & pediatric pts (≥15kg) with ataxia-telangiectasia (A-T) Approval was backed by the P-III (IB1001-303) trial assessing Aqneursa vs PBO in 73 pts (4-50yrs.) with genetically confirmed A-T, which met its 1 & key 2EPs & showed that Aqneursa has a…

ByRidhi Rastogi2 days ago

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Organon Reports the US FDA sNDA Acceptance for Miudella

Shots: The US FDA has accepted the sNDA of Miudella (hormone-free copper intrauterine system) for an extended duration of use for up to 6yrs. with the PDUFA action date expected in Q2’27 Miudella was evaluated in 3 trials across US in 1904 women (17-45yrs.), where the efficacy cohort (17-35yrs.) of the P-III trial (n=1397) showed…

ByRidhi RastogiSeptember 15, 2026

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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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Arrowhead Reports P-III (SHASTA-3 & 4) Trial Data on Redemplo (Plozasiran) for Severe Hypertriglyceridemia at ESC’26

Shots: P-III (SHASTA-3 & 4) trials, assessing Redemplo (25mg, SC, Q3M) vs PBO in sHTG adults (n=757), met their 1 & 2EPs, showing median TG reductions of 79% & 81%, respectively, at 12mos. In pts with TG ≥880mg/dL, median TG reductions were 85% in both trials At 12mos., Redemplo enabled 91% & 93% pts in…

ByRidhi RastogiAugust 31, 2026

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Celcuity Reports the US FDA sNDA Submission for Revtorpyk for HR+/HER2-, PIK3CA Mutant Breast Cancer

Shots: The US FDA has received sNDA for Revtorpyk (gedatolisib) for the treatment of pts with HR+, HER2-, locally advanced or metastatic breast cancer with a PIK3CA mutation, following progression on or after treatment with ≥1L of endocrine therapy sNDA was supported by the PIK3CA mutant cohort data from P-III (VIKTORIA-1) study of Revtorpyk + fulvestrant ±…

ByRidhi RastogiAugust 27, 2026

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Arrowhead Reports P-III (SHASTA-3 & SHASTA-4) Results on Plozasiran in Severe Hypertriglyceridemia (sHTG)

Shots: Arrowhead has reported topline P-III (SHASTA-3 & 4) trial results assessing plozasiran (25mg, SC, Q3M) vs PBO in sHTG pts (n=~750), where both trials met their 1EP of reduced median TGs by 79% & 81%, respectively, at 12mos. vs ~27% Pooled analyses showed significant reductions in AP event rates & total incidence rate, with…

ByRidhi RastogiJuly 23, 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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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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The US FDA Accepts Gilead’s Once-Weekly Oral Yeztugo for Review as a PrEP Therapy for HIV Prevention

Shots: The US FDA has accepted sNDA of Yeztugo (300mg, QW, PO) as a pre-exposure prophylaxis therapy for the prevention of HIV (PDUFA: Feb 02, 2027) sNDA was supported by the P-III (PURPOSE 1 & PURPOSE 2) trials of Yeztugo, which demonstrated high efficacy across diverse global populations, incl. cisgender women, cisgender men & gender-diverse…

ByRidhi RastogiJune 16, 2026

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Junshi Biosciences Reports P-III (NEOTORCH) Trial Results on Toripalimab + CT for Resectable Stage II-III NSCLC

Shots: Junshi Biosciences has reported the final analysis from the P-III (NEOTORCH) trial assessing toripalimab + Pt-containing doublet CT vs PBO + CT as perioperative treatment in 501 pts with resectable stage II-III NSCLC pts Trial improved EFS, with mEFS not reached vs 15.1mos. for CT alone, reducing risk of recurrence, progression, or death by 60%,…

ByRidhi RastogiMay 27, 2026

IntraBio Reports the US FDA Approval of Aqneursa for Ataxia-Telangiectasia

Organon Reports the US FDA sNDA Acceptance for Miudella

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

Arrowhead Reports P-III (SHASTA-3 & 4) Trial Data on Redemplo (Plozasiran) for Severe Hypertriglyceridemia at ESC’26

Celcuity Reports the US FDA sNDA Submission for Revtorpyk for HR+/HER2-, PIK3CA Mutant Breast Cancer

Arrowhead Reports P-III (SHASTA-3 & SHASTA-4) Results on Plozasiran in Severe Hypertriglyceridemia (sHTG)

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

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

The US FDA Accepts Gilead’s Once-Weekly Oral Yeztugo for Review as a PrEP Therapy for HIV Prevention

Junshi Biosciences Reports P-III (NEOTORCH) Trial Results on Toripalimab + CT for Resectable Stage II-III NSCLC

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