Rhythm Pharmaceuticals Reports Topline Data from P-III (TRANSCEND) Trial of Setmelanotide for Acquired Hypothalamic Obesity

Shots: The P-III (TRANSCEND) trial (N=120) assessed setmelanotide (n=81: 33 adults & 48 pediatric) vs PBO (n= 39) for acquired hypothalamic obesity in 49 adults & 71 pediatric pts; additional arm of 12 Japanese pts is ongoing, with topline data expected by Q1’26. FDA & EMA filings to be completed by Q3’25 Trial met its…

ByRidhi RastogiApril 8, 2025

NEWS

Teva Pharmaceuticals Reports the US FDA’s sBLA Acceptance of Ajovy (Fremanezumab) for Pediatric Episodic Migraine Prevention

Shots: The US FDA has accepted sBLA of Ajovy to prevent episodic migraine in children & adolescents (6-17yrs.; ≥45Kg) Submission was based on P-III (SPACE) trial assessing Ajovy (SC) vs PBO for 12wks. to treat episodic migraine in 237 pediatric pts, which showed improvements in reducing monthly migraine & headache days Ajovy (CGRP antagonist) is…

ByRidhi RastogiApril 8, 2025

NEWS

Johnson & Johnson Receives the EC’s Approval for Darzalex Regimen to Treat Newly Diagnosed Multiple Myeloma (NDMM)

Shots: The EC has approved Darzalex (SC) + bortezomib, lenalidomide & dexamethasone (D-VRd) to treat NDMM pts; sBLA under the US FDA’s review Approval was based on P-III (CEPHEUS) trial assessing D-VRd (n=197) vs VRd (n=198) in 395 ASCT-ineligible or deferred pts, in 13 countries across the EU, North & South America At 59mos. mFU, trial…

ByRidhi RastogiApril 8, 2025

NEWS

Intellia Therapeutics Reports the First Patient Dosing in P-III (MAGNITUDE-2) Trial of Nexiguran Ziclumeran for ATTRv-PN

Shots: Intellia has reported the first pts dosing in its P-III (MAGNITUDE-2) trial assessing nexiguran ziclumeran (nex-z) for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN); BLA submission is expected in 2028 Trial will assess nex-z (55mg infusion) vs PBO in ~50 ATTRv-PN pts to evaluate modified neuropathy impairment score & change in serum TTR levels as…

ByRidhi RastogiApril 7, 2025

NEWS

Amgen’s Uplizna Receives the US FDA’s Approval for Immunoglobulin G4-related Disease (IgG4-RD)

Shots: The US FDA has approved Uplizna to treat adults with IgG4-RD; regulatory filings for gMG are underway, with completion expected in H1’25 Approval was based on P-III (MITIGATE) trial assessing Uplizna (n=68) vs PBO (n=67) in IgG4-RD adults, which incl. an optional 3yr. OLE & up to 2yrs. of safety follow-up after Uplinza discontinuation Trial…

ByRidhi RastogiApril 4, 2025

NEWS

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

Shots: The US FDA has granted IND clearance to SNUG01 for ALS SNUG01 will be evaluated in a P-I/IIa dose-escalation & expansion trial to assess its safety, tolerability, & preliminary efficacy in ALS adults; SineuGene to partner with academics & institutions to lead clinical development of SNUG01 SNUG01, a TRIM72-targeted gene therapy, counteracts ALS through…

ByRidhi RastogiApril 3, 2025

NEWS

Novartis’ Vanrafia (Atrasentan) Receives the US FDA’s Accelerated Approval for Primary IgA Nephropathy (IgAN)

Shots: The US FDA has granted accelerated approval to Vanrafia for proteinuria reduction in adults with primary IgAN at risk of rapid disease progression based on the ongoing P-III (ALIGN) trial, with no requirement for REMS program; traditional approval will be supported by eGFR results expected in 2026 Trial assessed atrasentan (0.75mg, PO, QD) +…

ByDipanshu DixitApril 3, 2025

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The US FDA Grants 510(k) Clearance to Teleflex’s AC3 Range Intra-Aortic Balloon Pump (IABP) for Cardiac Patient Mobility

Shots: The US FDA has granted 510(k) clearance to the AC3 Range IABP, which will enter full market release in the US & begin shipping in Q2’25 AC3 Range allows reliable IABP support during multiple pts transport modes, incl. ambulances & fixed or rotary-wing aircraft AC3 Range integrates the AC3 Optimus IABP’s interface & algorithms…

ByRidhi RastogiApril 2, 2025

NEWS

Apellis Pharmaceuticals Reports the US FDA’s sNDA Acceptance & Priority Review of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Shots: The US FDA has accepted sNDA & granted priority review to Empaveli for C3 glomerulopathy (C3G) & primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) pts (PDUFA: Jul 28, 2025) Submission was backed by 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…

ByRidhi RastogiApril 2, 2025

NEWS

Alnylam Pharmaceuticals and Sanofi Report the US FDA’s Approval of Qfitlia (Fitusiran) to Treat Hemophilia A or B

Shots: The US FDA has approved Qfitlia to treat routine prophylaxis and prevent or reduce the frequency of bleeding episodes in pts. (age≥ 12yrs.) with hemophilia A or B, with or without factor VIII or IX inhibitors. Regulatory submissions have been completed in China & Brazil Qfitlia published clinical data in the NEJM in 2017, showing…

ByRidhi RastogiApril 1, 2025

Rhythm Pharmaceuticals Reports Topline Data from P-III (TRANSCEND) Trial of Setmelanotide for Acquired Hypothalamic Obesity

Teva Pharmaceuticals Reports the US FDA’s sBLA Acceptance of Ajovy (Fremanezumab) for Pediatric Episodic Migraine Prevention

Johnson & Johnson Receives the EC’s Approval for Darzalex Regimen to Treat Newly Diagnosed Multiple Myeloma (NDMM)

Intellia Therapeutics Reports the First Patient Dosing in P-III (MAGNITUDE-2) Trial of Nexiguran Ziclumeran for ATTRv-PN

Amgen’s Uplizna Receives the US FDA’s Approval for Immunoglobulin G4-related Disease (IgG4-RD)

SineuGene Therapeutics Reports the US FDA’s IND Clearance of SNUG01 for Amyotrophic Lateral Sclerosis

Novartis’ Vanrafia (Atrasentan) Receives the US FDA’s Accelerated Approval for Primary IgA Nephropathy (IgAN)

The US FDA Grants 510(k) Clearance to Teleflex’s AC3 Range Intra-Aortic Balloon Pump (IABP) for Cardiac Patient Mobility

Apellis Pharmaceuticals Reports the US FDA’s sNDA Acceptance & Priority Review of Empaveli for C3 Glomerulopathy and Primary IC-MPGN

Alnylam Pharmaceuticals and Sanofi Report the US FDA’s Approval of Qfitlia (Fitusiran) to Treat Hemophilia A or B

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