Mirum Reports Topline P-III (EXPAND) Trial Data on Livmarli (Maralixibat) in Ultra-Rare Cholestatic Liver Diseases

Shots: The P-III (EXPAND) trial assessed Livmarli (285µg/kg, BID,PO) vs PBO for cholestatic pruritus in pts (≥6mos.; n=52: 6 adults & 46 pediatric) with ultra-rare cholestatic liver diseases, incl. biliary atresia (BA), achieving statistically significance across all 5 EPs In the pediatric population, Livmarli improved pruritus severity (ItchRO(Obs): −1.81 vs −0.83) & increased days with…

ByRidhi Rastogi5 hours ago

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Bayer Reports US FDA sNDA Acceptance for Kerendia to Treat Non-Diabetic CKD

Shots: The US FDA has accepted sNDA of Kerendia (finerenone) for the treatment of adults with chronic kidney disease (CKD) without diabetes sNDA was supported by the P-III (FIND-CKD) trial assessing Kerendia (10 or 20mg) vs PBO, both on top of maximum tolerated labeled doses of a RAS-blocking therapy, in >1,500 adults with non-diabetic CKD …

ByRidhi RastogiOctober 8, 2026

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Vanda Pharmaceuticals Reports the P-III Trial Results on Hetlioz in Delayed Sleep-Wake Phase Disorder

Shots: Vanda Pharmaceuticals has reported the P-III (VP-VEC-162-3502) trial data assessing Hetlioz (tasimelteon; 20mg, PO, QD; n=20) vs PBO (n=20) in 43 adults with Delayed Sleep-Wake Phase Disorder (DSWPD) Trial met its 1EP, showing Hetlioz (20mg) shifted the time of sleep onset 42.5 min. earlier vs 5.4 min. shift on PBO, plus in an exploratory…

ByRidhi RastogiSeptember 30, 2026

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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 RastogiSeptember 21, 2026

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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

Mirum Reports Topline P-III (EXPAND) Trial Data on Livmarli (Maralixibat) in Ultra-Rare Cholestatic Liver Diseases

Bayer Reports US FDA sNDA Acceptance for Kerendia to Treat Non-Diabetic CKD

Vanda Pharmaceuticals Reports the P-III Trial Results on Hetlioz in Delayed Sleep-Wake Phase Disorder

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

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