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Results For "Orphan-Drug-designations"

13 News Found

GSK bets big on oncology with $10.6 billion Nuvalent acquisition
News | June 10, 2026

GSK bets big on oncology with $10.6 billion Nuvalent acquisition

Deal brings late-stage lung cancer candidates zidesamtinib and neladalkib into GSK’s portfolio


SN Bioscience doses first patient in global trial of experimental drug for aggressive lung cancer
Clinical Trials | May 19, 2026

SN Bioscience doses first patient in global trial of experimental drug for aggressive lung cancer

The drug also delivered a disease control rate (DCR) of 83.3% in high-dose cohorts and a mean progression-free survival of 6.3 months in SCLC patients


EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease
Drug Approval | December 20, 2025

EMA grants orphan drug status to Sanofi’s Efdoralprin Alfa for rare lung disease

Efdoralprin alfa, a recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein, showed superior efficacy to standard plasma-derived therapy in adults with AATD


Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder
News | December 19, 2025

Ambros Therapeutics launches with $125 million to advance breakthrough therapy for rare pain disorder

The funding will support the pivotal Phase 3 clinical trial of neridronate in CRPS-1


Bayer advances fight against rare kidney disease with new experimental drug
R&D | December 07, 2025

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes


FDA grants accelerated approval to sevabertinib for non-squamous non-small cell lung cancer
Drug Approval | November 21, 2025

FDA grants accelerated approval to sevabertinib for non-squamous non-small cell lung cancer

HER2-mutant NSCLC typically has a poor prognosis and limited treatment options


Ipsen to acquire ImCheck Therapeutics to strengthen oncology pipeline
News | October 23, 2025

Ipsen to acquire ImCheck Therapeutics to strengthen oncology pipeline

The acquisition centers on ImCheck’s lead Phase I/II program, ICT01, a first-in-class monoclonal antibody targeting BTN3A


FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia
Drug Approval | September 02, 2025

FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia

ITP is a disease of complex immune dysregulation leading to low platelet counts, bleeding, and reduced quality of life


Zydus Lifesciences reports Q1 FY26 net profit at Rs. 1,466.8 Cr
News | August 13, 2025

Zydus Lifesciences reports Q1 FY26 net profit at Rs. 1,466.8 Cr

The company reported revenue from operations at Rs. 6,573.7 crore, up 6 per cent YoY


Sentynl Therapeutics receives USFDA acceptance and priority review of new drug application for CUTX-101 product candidate for treatment of Menkes disease
Drug Approval | January 07, 2025

Sentynl Therapeutics receives USFDA acceptance and priority review of new drug application for CUTX-101 product candidate for treatment of Menkes disease

Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A