Record number of orphan designated medicines recommended for approval in 2014

By William Chin, PhD, Scientific Coordinator, EUCRAF

The year 2014 saw the highest number of orphan designated medicines granted approval for marketing authorisation. 21% of the total approved medicines (17/82) are intended for the treatment of a rare disease. EMA reported that the agency provided more scientific support in the early stages of medicine development in 2014 - almost seven out of ten applicants received scientific advice from EMA’s CHMP during the development phase of their medicine and this figure rises to four out of five when it comes to innovative medicines. Here are the list of orphan designated medicines approved in 2014:

Medicine Active Substance MAH Authorisation date Indication
Lynparza olaparib AstraZeneca AB 16/12/2014 Lynparza is indicated as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed BRCA-mutated (germline and/or somatic) high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer
Imbruvica ibrutinib Janssen-Cilag International NV 21/10/2014 Imbruvica is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL)
Translarna ataluren PTC Therapeutics International Limited 31/07/2014 Translarna is indicated for the treatment of Duchenne muscular dystrophy
Gazyvaro obinutuzumab Roche Registration Ltd 23/07/2014 Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia
Sylvant siltuximab Janssen-Cilag International NV 22/05/2014 Sylvant is indicated for the treatment of adult patients with multicentric Castlemans disease
Deltyba delamanid Otsuka Novel Products GmbH 28/04/2014 Deltyba is indicated for use as part of an appropriate combination regimen for pulmonary multi-drug resistant tuberculosis (MDR-TB) in adult patients
Vimizim recombinant human n-acetylgalactosamine-6-sulfatase (rhgalns) BioMarin Europe Ltd 28/04/2014 Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages.
Granupas (previously Para-aminosalicylic acid Lucane) para-aminosalicylic acid Lucane Pharma 07/04/2014 Granupas is indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients
Kolbam (previously Cholic Acid FGK)
cholic acid
FGK Representative Service GmbH 04/04/2014 Kolbamis indicated for the treatment of inborn errors in primary bile acid synthesis in infants, children and adolescents aged 1 month to 18 years and adults.
Adempas riociguat Bayer Pharma AG 27/03/2014 Chronic thromboembolic pulmonary hypertension (CTEPH) Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with inoperable CTEPH, persistent or recurrent CTEPH after surgical treatment
Cometriq cabozantinib TMC Pharma Services Ltd 21/03/2014 Treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma
Sirturo bedaquiline fumarate Janssen-Cilag International N.V. 05/03/2014 Indicated for use as part of an appropriate combination regimen for pulmonary multidrug resistant tuberculosis (MDR TB) in adult patients when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. 

Source

1.EMA, Press Release, Record number of medicines for rare diseases recommended for approval in 2014, 9 January 2015