Orphan Drugs
NMD Pharma’s Charcot-Marie-Tooth disease treatment granted ODD by FDA
January 6, 2025CMT, FDA, NMD Pharma, Orphan Drugs, Pain, nervous system, research and development
Biotech company NMD Pharma has announced that the US Food and Drug Association (FDA) have granted its Charcot-Marie-Tooth (CMT) disease …
Global orphan and rare dermatology market set to exceed $6 billion by 2024
August 31, 2018Medical Communications Orphan Drugs, analysis, dermatology, market reports, rare diseases
The global orphan and rare dermatology premium products market is expected to grow rapidly in the next half a decade …
FDA approves RNA therapy for rare genetic disease
August 13, 2018Sales and Marketing FDA, Onpattro, Orphan Drugs, RNA, rare diseases
The US Food and Drug Administration has approved the RNA-based therapy Onpattro, for the treatment of the rare genetic disease …
TREND: Rare disease, cancer drugs will drive pharma M&A’s, growth
March 29, 2016Manufacturing and Production, Research and Development, Sales and Marketing M&A, MA, Orphan Drugs, cancer drugs
Moody’s Investor Service lowered its growth outlook for the global pharma sector but maintained the mergers and acquisitions in the …
EMA recommends AZ/Acerta drug for orphan status in three indications
February 25, 2016Research and Development AstraZeneca, EMA, Orphan Drugs
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended AstraZeneca and Acerta’s acalabrutinib (ACP-196) for designation …
Novartis’ myelofibrosis treatment approved in Europe
August 28, 2012Sales and Marketing Incyte, Jakavi, Novartis, Orphan Drugs, myelofibrosis
Novartis’ Jakavi, a new treatment for patients with myelofibrosis has been approved in Europe. The drug was co-developed with Incyte, …
First idiopathic pulmonary fibrosis drug approved
March 4, 2011Sales and Marketing Esbriet, IPF, InterMune, Orphan Drugs, idiopathic pulmonary fibrosis, orphan drug
The first ever treatment for the potentially fatal lung disease idiopathic pulmonary fibrosis (IPF) has been approved in the EU. …
Genzyme again snubs suitor Sanofi
November 10, 2010Research and Development, Sales and Marketing Campath, Chris Viehbacher, Genzyme, Henri Termeer, Orphan Drugs, Plavix, Sanofi, Sanofi-Aventis, manufacturing compliance
Genzyme has once again snubbed Sanofi-Aventis as the French pharma firm continues its pursuit of the beleaguered biotech company. Henri …
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