Early access and regulation

Novartis orphan drug wins 'breakthrough' status from FDA
Novartis orphan drug wins 'breakthrough' status from FDA Bimagrumab could be first treatment for rare life-threatening muscle-wasting condition
FDA panel turns down Otsuka’s drug for rare kidney disease
FDA panel turns down Otsuka’s drug for rare kidney disease Unable to recommend expanded use of tolvaptan
BTG extends orphan drug distribution partnership with Clinigen
Deal covers treatment for overexposure to chemotherapy
Actelion to buy Ceptaris - if FDA backs its cancer treatment
Actelion to buy Ceptaris - if FDA backs its cancer treatment Acquisition contingent on Valchlor approval in rare lymphoma
Enabling access for patients with rare diseases
Enabling access for patients with rare diseases When it comes to meeting patient needs, the well-known counsel to ‘think globally, act locally’ rings true
EC consults on orphan drug application guidelines
Also seeks input on the transfer of designations from one sponsor to another
Court orders EC to approve rejected orphan medicine
Court orders EC to approve rejected orphan medicine Says CTRS should be allowed to market Orphacol for the treatment of rare bile condition
FDA grants orphan status to Activartis’ brain cancer immunotherapy
FDA grants orphan status to Activartis’ brain cancer immunotherapy Regulator says AV0113 has potential in rare cancer malignant glioma
Celgene wins expanded EU approval for cancer drug Revlimid
Celgene wins expanded EU approval for cancer drug Revlimid Approved for use in patients with rare blood disorder
Celgene's Revlimid wins US lymphoma approval
Celgene's Revlimid wins US lymphoma approval FDA recommends extra indication for blockbuster cancer drug
FDA gives priority review status to BMS/ AZ's metreleptin
FDA gives priority review status to BMS/ AZ's metreleptin Former obesity prospect has potential in the rare disease lipodystrophy
FDA gives Alexion's metabolic drug breakthrough status
FDA gives Alexion's metabolic drug breakthrough status Reduces requirements for registration, potentially speeding up approval
Orphan status in US for Astellas/ Basilea antifungal
Orphan status in US for Astellas/ Basilea antifungal Isavuconazole is being developed as a treatment for invasive aspergillosis
Novartis gets FDA green light for new Ilaris indication
Novartis gets FDA green light for new Ilaris indication Immunotherapeutic wins US approval for severe form of childhood arthritis
FDA approves drugs for rare genetic liver condition and to reverse anticoagulation
FDA approves drugs for rare genetic liver condition and to reverse anticoagulation Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US
European drug applications 'stable but more complex in 2012'
European drug applications 'stable but more complex in 2012' Despite austerity measures, EMA's annual report shows signs of biopharma resilience
Otsuka files new TB candidate in Japan as shortages worsen
Otsuka files new TB candidate in Japan as shortages worsen Delamanid shows promise in multi-drug resistant TB but supplies of rifampin are low
Takeda files lymphoma drug brentuximab vedotin in Japan
Takeda files lymphoma drug brentuximab vedotin in Japan Seeks approval for antibody-drug conjugate for two forms of the blood cancer
FDA says approval of generic Doxil will ease cancer drug shortage
FDA says approval of generic Doxil will ease cancer drug shortage Allows Sun Pharma to manufacture its own version of the medicine
US approval for Sanofi’s Kynamro in genetic cholesterol disorder
US approval for Sanofi’s Kynamro in genetic cholesterol disorder Comes one month after drug was turned down in Europe due to safety concerns

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