Tag: Rare Disease

NICE Published Amended Draft Guidance for Spinraza

More children with the rare genetic disorder spinal muscular atrophy (SMA) can now be treated with Spinraza after on 3 July NICE published amended draft guidance following a proposal for extending t...

Sobi Buys Rare-Disease Drug Emapalumab

Rare diseases specialist Swedish Orphan Biovitrum (Sobi) has agreed to buy the drug emapalumab and is reorganizing to increase focus on late-stage development in hematology and immunology. The drug...

BTD for AstraZeneca and Merck Med

AstraZeneca and MSD, Inc., Kenilworth, NJ, US announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the MEK 1/2 inhibitor and potential new me...

FDA Guidance to Support Development of Rare Diseases Treatments

fda office
The US Food and Drug Administration (FDA) published new draft guidance to help inform the design and implementation of natural history studies that can be used to support the development of treatments...

Alexion and Affibody Partner to Develop Rare Disease Treatment

Alexion
Alexion Pharmaceuticals, Inc. and Affibody AB announced a partnership to co-develop ABY-039 for rare Immunoglobulin G (IgG)-mediated autoimmune diseases. Currently in Phase 1 development, ABY-039 is a...

Pfizer and Vivet Collaborate to Fight Rare Disease

Vivet Therapeutics, a privately held gene therapy biotech company dedicated to developing gene therapy treatments for inherited liver disorders with high unmet medical need, and Pfizer Inc. announced ...

Canadian Government Promises to Build Natonal Drug Agency

The federal Liberal government is promising a new agency to negotiate prescription drug prices for Canadians and try to drive down costs — a move billed as an "important step" on the path to an eventu...

Boehringer Ingelheim Seeks Approval of FDA and EMA for SSc-ILD Drug

Boehringer Ingelheim has filed for regulatory approval of nintedanib in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD) with the FDA and EMA. Nintedanib as an anti-fibr...

AstraZeneca New Med for Rare Disease Got ODD

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for saracatinib, a potential new medicine for the treatment of idiopathic pulmonary fibrosis (IPF), a type of lung d...

European Approval for Roche’s Treatment for Rare Disease

Roche today announced that the European Commission has approved MabThera® (rituximab) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), a rare condition characterised by pro...

EMA Recommended First Med for FCS

EMA
The European Medicines Agency has recommended granting a conditional marketing authorisation (CMA) for Waylivra (volanesorsen), the first medicine for the treatment of the familial chylomicronaemia sy...

Priority Review for Alexion’s Rare Disease Drug

Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental Biologics License Application (sBLA) for the use of SOLIRIS® (eculizumab...

NICE Declined Brineura Again

NICE has published draft guidance which confirms its earlier decision not to recommend cerliponase alfa (also called Brineura and made by Biomarin) for children with Batten disease – a very rare inher...

ODD for Ocugen’s Novel Gene Therapy

Ocugen, Inc., a clinical stage biopharmaceutical company focused on discovering, developing and commercializing a pipeline of innovative therapies that address rare and underserved eye diseases, today...

Preferential VAT Policy for Rare Diseases in China

China will offer preferential value-added tax (VAT) policies for drugs aimed at the treatment of rare diseases, a State Council executive meeting chaired by Premier Li Keqiang decided on Monday. Fr...

First Drug for Rare Cancer under Priority Review

Daiichi Sankyo has been given a priority review from the FDA for pexidartinib, which is vying to become the first systemic drug therapy for rare cancer tenosynovial giant cell tumour (TGCT). The Ja...