Novo Nordisk’s Alhemo Receives the US FDA’s Approval for Hemophilia A or B (HA/HB) Without Inhibitors

Shots: The US FDA has approved label expansion of Alhemo (concizumab-mtci) to prevent or reduce the frequency of bleeding episodes in pts (≥12yrs.) with HA/HB without inhibitors Approval was based on P-III (Explorer8) trial assessing Alhemo (n=42, 1mg/kg followed by 0.2mg/kg QD) vs no prophylaxis (n=21) in 156 males with HA/HB, where median duration of…

ByRidhi RastogiAugust 1, 2025

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LENZ Therapeutics’ Vizz Receives the US FDA’s Approval to Treat Presbyopia

Shots: The US FDA has approved Vizz (1.44% aceclidine ophthalmic solution) for the treatment of presbyopia in adults; samples are expected in the US by Oct 2025, with full commercial launch by mid-Q4’25 Approval was based on the 3 P-III trials: CLARITY 1 & 2 evaluating Vizz (QD) in 466 pts for 42 days as…

ByRidhi RastogiAugust 1, 2025

NEWS

AbbVie Reports the US FDA’s sNDA Submission of Venclexta + Acalabrutinib for Chronic Lymphocytic Leukemia

Shots: The US FDA has received sNDA of Venclexta (venetoclax) & acalabrutinib in previously untreated patients with chronic lymphocytic leukemia (CLL) sNDA was supported by AstraZeneca-sponsored P-III (AMPLIFY) trial assessing Venclexta + acalabrutinib ± Obinutuzumab vs chemoimmunotherapy in previously untreated CLL pts without del(17p) or TP53 mutation Trial showed that Venclexta + acalabrutinib a 35% reduction…

ByRidhi RastogiJuly 31, 2025

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Purpose Pharma’s Attrogy Secures the EC’s Approval for the Treatment of Hereditary Transthyretin-mediated Amyloidosis

Shots: The EC has approved Attrogy (diflunisal) to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or 2 polyneuropathy in all EEA states EC approval was based on a P-III trial showing that diflunisal significantly slowed disease progression in patients with TTR amyloidosis. At 24 mos. baseline, 1EP met diflunisal led to…

ByDipanshu DixitJuly 31, 2025

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IntraBio’s Aqneursa Receives the CHMP’s Positive Opinion for Treating Niemann-Pick Disease Type C (NPC)

Shots: The CHMP has recommended Aqneursa (levacetylleucine) for treating adults & pediatric pts with NPC; additional global submissions are planned for 2025 & beyond Approval was backed by P-III (IB1001-301) study assessing Aqneursa vs PBO in pts (≥4yrs.; n=60) with NPC, which showed improved neurological symptoms & function across all 1 & 2EPs within 12wks.,…

ByRidhi RastogiJuly 30, 2025

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Apellis Pharmaceuticals Reports the US FDA’s Approval of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Shots: The US FDA has approved Empaveli (pegcetacoplan) for C3 glomerulopathy (C3G) & primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) pts (≥12yrs.) Approval was based on P-III (VALIANT) trial assessing Empaveli (Q2W) vs PBO in C3G & primary IC-MPGN pts (≥12yrs., n=124) for 26wks. followed by 26wk. open-label period, where all pts received Empaveli Trial met…

ByRidhi RastogiJuly 30, 2025

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Deciphera Pharmaceuticals Reports the CHMP’s Positive Opinion of Romvimza for Symptomatic Tenosynovial Giant Cell Tumor (TGCT)

Shots: The CHMP has recommended Romvimza (vimseltinib) to treat symptomatic TGCT with physical function deterioration in adults for which surgery may lead to functional impairment or severe morbidity; EC’s decision is expected in Q3'25 Opinion was based on P-I/II trial as well as P-III (MOTION) trial, which evaluated Romvimza vs PBO in surgery-ineligible pts without…

ByRidhi RastogiJuly 29, 2025

NEWS

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Shots: The US FDA has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU) in pts of all ages, with broad labeling for hyperphenylalaninemia in adults & pediatric pts (≥1mos.) with sepiapterin-responsive PKU; review is ongoing in Japan & Brazil Approval was based on APHENITY study results & long-term extension data showing durable effects and…

ByRidhi RastogiJuly 29, 2025

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Ionis Reports the CHMP’s Positive Opinion on Tryngolza for Familial Chylomicronemia Syndrome

Shots: The CHMP has recommended Tryngolza (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome; EC’s decision is expected in Q4’25 Opinion was based on P-III (Balance) trial assessing (Q4W) vs PBO, which showed reduced triglyceride levels at 6mos., sustained through 12mos., with decrease in acute…

ByRidhi RastogiJuly 29, 2025

NEWS

Sobi Reports the US FDA’s Approval of Doptelet (Avatrombopag) to Treat Pediatric Immune Thrombocytopenia

Shots: The US FDA has approved Doptelet to treat thrombocytopenia in pts (≥1yrs.) with persistent or chronic ITP who have not responded to prior treatments. Approval also incl. a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for children (1-6yrs.) Approval was based on P-III (AVA-PED-301) study assessing Doptelet in pediatric ITP pts, which showed that…

ByRidhi RastogiJuly 28, 2025

Novo Nordisk’s Alhemo Receives the US FDA’s Approval for Hemophilia A or B (HA/HB) Without Inhibitors

LENZ Therapeutics’ Vizz Receives the US FDA’s Approval to Treat Presbyopia

AbbVie Reports the US FDA’s sNDA Submission of Venclexta + Acalabrutinib for Chronic Lymphocytic Leukemia

Purpose Pharma’s Attrogy Secures the EC’s Approval for the Treatment of Hereditary Transthyretin-mediated Amyloidosis

IntraBio’s Aqneursa Receives the CHMP’s Positive Opinion for Treating Niemann-Pick Disease Type C (NPC)

Apellis Pharmaceuticals Reports the US FDA’s Approval of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Deciphera Pharmaceuticals Reports the CHMP’s Positive Opinion of Romvimza for Symptomatic Tenosynovial Giant Cell Tumor (TGCT)

PTC Therapeutics Receives the US FDA’s Approval for Sephience to Treat Phenylketonuria

Ionis Reports the CHMP’s Positive Opinion on Tryngolza for Familial Chylomicronemia Syndrome

Sobi Reports the US FDA’s Approval of Doptelet (Avatrombopag) to Treat Pediatric Immune Thrombocytopenia

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