NEWS

Sanofi’s Venglustat Secures the US FDA Breakthrough Therapy Designation for Type 3 Gaucher Disease

Shots: The US FDA has granted BTD to venglustat for the treatment of neurological manifestations of type 3 Gaucher disease (GD3) Designation was based on the P-III (LEAP2MONO) study assessing venglustat (QD, PO) vs imiglucerase (Q2W) in 43 pts (≥12yrs.), which showed significant improvements in neurological symptoms measured by a global test scores, incl. mSARA & RBANS Venglustat received US…

10 hours ago

Myriad Genetics Receives the US FDA Approval for MyChoice CDx Test as a Companion Diagnostic for GSK’s Zejula

Shots: The US FDA has approved MyChoice CDx Test as a companion diagnostic for for HRD+ identification in pts with advanced ovarian cancer who may benefit from treatment with Zejula (niraparib) Approval was based on the PRIMA trial assessing Zejula vs PBO in ovarian cancer pts with complete or partial response to 1L Pt-based CT,…

11 hours ago

Samsung Bioepis Partners with Sandoz for Up to Five Biosimilar Candidates

Shots: Samsung Bioepis has entered into a global license, development & commercialization agreement with Sandoz for up to five biosimilars, incl. SB36 (preclinical), a biosimilar version of Entyvio (vedolizumab) Samsung Bioepis will handle development, manufacturing & regulatory filings, while Sandoz will lead commercialization globally (excl. China, Hong Kong, Taiwan, Macau & Republic of Korea) The…

12 hours ago

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…

14 hours ago

Ascendis Pharma Reports Topline P-II (New InsiGHTS) Trial Data on TransCon hGH in Turner Syndrome

Shots: Ascendis Pharma has reported the P-II (New InsiGHTS) trial data assessing TransCon hGH (lonapegsomatropin, QW) vs somatropin (QD) in 49 prepubertal children (1-10yrs.) with Turner syndrome At Wk. 52, TransCon hGH showed improved annualized height velocity (AHV) with LS mean AHV of 9.05 vs 9.04 cm/year, irrespective of starting dose, with mean dose of…

15 hours ago

Voro Therapeutics Collaborates with Daiichi Sankyo to Develop Tumor-Activated ADCs

Shots: Voro Therapeutics has entered into a research collaboration with Daiichi Sankyo via its Daiichi Sankyo Research Institute, San Diego to develop tumor-activated biologics using the PrimeBody platform The collaboration will focus on generating masked ADCs designed for tumor-selective activation, using proprietary masking domains & protease-cleavable linkers to improve therapeutic index Voro will design &…

16 hours ago

Pfizer Reports P-II (FOURLIGHT-1) Trial Data on Atirmociclib Combination for 2L Metastatic Breast Cancer

Shots: Pfizer has reported P-II (FOURLIGHT-1) trial data assessing atirmociclib + fulvestrant vs fulvestrant or everolimus plus exemestane in 264 pts with HR+, HER2- advanced or metastatic breast cancer who had received prior CDK 4/6 inhibitor-based treatment Trial met its 1EP, showing improved PFS as assessed by the investigator, with benefit consistent across all subgroups. OS…

1 day ago

Sun Pharma Reports US FDA’s sBLA Acceptance of Ilumya for Active Psoriatic Arthritis

Shots: The US FDA has accepted sBLA of Ilumya (tildrakizumab-asmn) for the treatment of adults with active psoriatic arthritis (PsA), with PDUFA target action date of Oct 29, 2026 sBLA was backed by P-III (INSPIRE-1 & INSPIRE-2) trials assessing Ilumya (100mg) in pts with PsA over 52wks., where INSPIRE-1 enrolled pts with prior anti-TNF exposure…

1 day ago

AstraZeneca Secures the EC Approval of Imfinzi as a Perioperative Therapy for G/GEJ Cancers

Shots: The EC has approved perioperative Imfinzi + FLOT for adults with resectable, early-stage & locally advanced (Stages II, III, IVA) G/GEJ cancers, based on P-III (MATTERHORN) trial; regulatory review is ongoing in Japan In the trial, Pts (n=948) received either Imfinzi (1500mg) + FLOT or PBO + FLOT for Q4W × 2 cycles, then surgery followed by…

2 days ago

Celltrion Introduces Avtozma SC (Biosimilar, Actemra) in the US

Shots: Celltrion launched Avtozma SC, a biosimilar to Actemra (tocilizumab), in the US, becoming among the first to offer both IV & SC formulations approved by the US FDA & available in the market Avtozma received the FDA & EC approval in Jan & Feb 2025, respectively, based on a global P-III trial showing comparable…