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RSS FeedPosted by Michael Wonder on 30 Nov 2018
Vertex announces European Commission approval for Kalydeco (ivacaftor) to treat patients with cystic fibrosis aged 12 to <24 months with certain mutations in the CFTR gene
29 November 2018 - Ivacaftor is the first and only approved medicine in Europe to treat the underlying cause of cystic ...
Posted by Michael Wonder on 01 Nov 2018
Vertex announces European authorisation for third cystic fibrosis medicine Symkevi (tezacaftor/ivacaftor), to be used in combination with ivacaftor (Kalydeco), for people with CF aged 12 and older with certain mutations in the CFTR gene
1 November 2018 - First medicine in the EU to treat the CFTR protein defect in patients who have one copy ...
Posted by Michael Wonder on 28 Aug 2018
Ultragenyx announces approval of Mepsevii (vestronidase alfa) in Europe for the treatment of mucopolysaccharidosis VII
27 August 2018 - Mepsevii, an enzyme replacement therapy, is the first treatment approved in the EU for mucopolysaccharidosis VII. ...
Posted by Michael Wonder on 16 Aug 2018
FDA approves Kalydeco (ivacaftor) as first and only medicine to treat the underlying cause of CF in children ages 12 to <24 months with certain mutations in the CFTR gene
15 August 2018 - Data from Phase 3 ARRIVAL study support treatment with Kalydeco in children ages 12 to <24 months. ...
Posted by Michael Wonder on 31 Jul 2018
Alnylam receives positive CHMP opinion for Onpattro (patisiran) for the treatment of hereditary transthyretin-mediated amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy
27 July 2018 - European Commission decision expected in September. ...
Posted by Michael Wonder on 31 Jul 2018
Vertex receives European CHMP positive opinion for Symkevi (tezacaftor/ivacaftor) for people with cystic fibrosis aged 12 and older with certain mutations in the CFTR gene
27 July 2018 - If approved, Symkevi (tezacaftor/ivacaftor)will be Vertex’s third medicine to treat the CFTR protein defect in patients ...
Posted by Michael Wonder on 02 Jul 2018
Ultragenyx announces positive CHMP opinion for Mepsevii (vestronidase alfa) for the treatment of mucopolysaccharidosis VII
29 June 2018 - Ultragenyx today announced that the CHMP), the scientific committee of the EMA, has adopted a positive opinion ...
Posted by Michael Wonder on 28 Jun 2018
Alexion submits application for approval of ALXN1210 as a treatment for patients with paroxysmal nocturnal haemoglobinuria in the European Union
28 June 2018 - EU filing follows U.S. filing in June 2018. ...
Posted by Michael Wonder on 19 Jun 2018
Modernising the orphan designation process
19 June 2018 - The EMA has launched a new secure online portal for orphan designationExternal link icon applications. ...
Posted by Michael Wonder on 22 May 2018
Working together for people with rare and complex diseases
22 May 2018 - Live broadcast of workshop with European Reference Networks on 29 May. ...
Posted by Michael Wonder on 11 Apr 2018
Kineret (anakinra) approved in the EU for the treatment of Still’s disease
11 April 2018 - Sobi announces that the European Commission has approved an extension of the indication for Kineret (anakinra) ...
Posted by Michael Wonder on 05 Apr 2018
Chiesi Group receives the European marketing authorisation for Lamzede (velmanase alfa)
4 April 2018 - Lamzede (velmanase alfa) is the first enzyme replacement therapy for the treatment of non-neurological manifestations in patients ...
Posted by Michael Wonder on 29 Mar 2018
EMA accepts BioMarin's marketing application for pegvaliase MAA for treatment of phenylketonuria
28 March 2018 - BioMarin Pharmaceutical announced today that the EMA has accepted BioMarin's submission of a marketing authorisation application for ...
Posted by Michael Wonder on 26 Mar 2018
Alnylam receives EMA PRIME designation for accelerated assessment of lumasiran, an investigational RNAi therapeutic for the treatment of primary hyperoxaluria type 1
26 March 2018 - Company intends to advance lumasiran to a Phase 3 study in late 2018. ...
Posted by Michael Wonder on 26 Jan 2018
Alnylam Announces EMA acceptance of marketing authorisation application for patisiran for the treatment of hereditary ATTR amyloidosis
25 January 2018 - EMA will evaluate the application under accelerated assessment. ...