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Results For "rare-disease"

198 News Found

AstraZeneca 1Q 2024 revenue up 19% to US$ 12,679 million
News | April 27, 2024

AstraZeneca 1Q 2024 revenue up 19% to US$ 12,679 million

Very strong revenue and EPS growth in the first quarter coupled with exciting pipeline delivery


Sentynl Therapeutics receives marketing authorization for Nulibry from UK MHRA
Drug Approval | April 22, 2024

Sentynl Therapeutics receives marketing authorization for Nulibry from UK MHRA

NULIBRY is a first-in-class synthetic cPMP substrate replacement therapy to reduce the risk of mortality in patients with MoCD Type A


USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria
News | April 17, 2024

USFDA grants ODD to NM5072 for treatment of Paroxysmal Nocturnal Hemoglobinuria

NM5072 is an anti-Properdin antibody that selectively blocks the alternative pathway while maintaining the integrity of the classical pathway required for fending off infections


FDA Granted ODD to Utidelone Injectable from Biostar Pharma for treatment of breast cancer brain metastasis
Drug Approval | April 01, 2024

FDA Granted ODD to Utidelone Injectable from Biostar Pharma for treatment of breast cancer brain metastasis

Utidelone has the ability to cross BBB due to its unique physicochemical characteristic and insusceptibility to P-glycoprotein-mediated efflux


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