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Results For "rare-disease"

234 News Found

Sanofi and Cipla enters into distribution partnership to expand reach of CNS portfolio in India
Supply Chain | March 27, 2024

Sanofi and Cipla enters into distribution partnership to expand reach of CNS portfolio in India

Cipla will be responsible for the distribution of Sanofi India's six CNS brands including Frisium


Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder
Drug Approval | March 27, 2024

Ultomiris approved in the US for the treatment of adults with neuromyelitis optica spectrum disorder

First and only long-acting C5 complement inhibitor offers patients with AQP4 Ab+


FDA approves Merck’s WINREVAIR for treatment of pulmonary arterial hypertension
Drug Approval | March 27, 2024

FDA approves Merck’s WINREVAIR for treatment of pulmonary arterial hypertension

WINREVAIR is a breakthrough biologic for this rare, progressive disease


USFDA grants ODD to Cevidoplenib for immune thrombocytopenia
News | March 23, 2024

USFDA grants ODD to Cevidoplenib for immune thrombocytopenia

Oscotec has successfully completed phase 2 study in patients with chronic ITP last year


AstraZeneca to acquire Amolyt Pharma
News | March 18, 2024

AstraZeneca to acquire Amolyt Pharma

Acquisition to further expand the Alexion, AstraZeneca Rare Disease pipeline beyond complement inhibition


Moderna posts Q4 2023 revenue at US$ 2.8 billion
News | February 23, 2024

Moderna posts Q4 2023 revenue at US$ 2.8 billion

Net product sales in the fourth quarter of 2023 include the recognition of $0.6 billion from deferred revenue, related to Gavi, the Vaccine Alliance


ImmunoBrain Checkpoint appoints Dr. Sanjay Keswani as CEO
People | January 24, 2024

ImmunoBrain Checkpoint appoints Dr. Sanjay Keswani as CEO

He is a former Neurology faculty member at The Johns Hopkins Hospital and an elected Fellow of the Royal College of Physicians, United Kingdom


EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome
Drug Approval | January 23, 2024

EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome

GC1130A has previously achieved notable milestones by securing both RPDD and ODD from the U.S. FDA in January 2023


European Commission grants ODD to NS-229 for treatment of Eosinophilic Granulomatosis
Clinical Trials | January 23, 2024

European Commission grants ODD to NS-229 for treatment of Eosinophilic Granulomatosis

The orphan drug designation by the EC is issued for drugs which are intended to treat diseases that affect fewer than five in 10,000 people in the European Union