Vertex Pharmaceuticals Inc has received a new approval from the US Food and Drug Administration for its cystic fibrosis treatment Kalydeco (ivacaftor) that triples the number of rare gene mutations that the drug can treat to 33 from 10.
In a reversal of the usual sequence of events, the Food and Drug Administration has approved a new drug for amyotrophic lateral sclerosis (ALS) two years after the same treatment was authorised for marketing in Japan and South Korea.
The European Medicines Agency has issued guidance to companies with centralised marketing authorisations spelling out what they need to do to be in compliance with EU medicines law once Britain leaves the union.
An enzyme replacement therapy, Brineura (cerliponase alfa), has been approved in the US for children with a rare inherited disorder that primarily affects the nervous system. The disorder, tripeptidyl peptidase-1 (TPP1) deficiency, is a form of Batten disease.
AstraZeneca Plc has received an accelerated approval in the US for its checkpoint antibody Imfinzi (durvalumab), which is expected to be the cornerstone of a prospective portfolio of immune oncology products targeting a variety of cancers.
Novartis has secured US approval for a new kinase inhibitor, Rydapt (midostaurin), to treat patients with FLT3-mutated acute myeloid leukaemia (AML) in combination with chemotherapy. The approval also covers a diagnostic designed to detect the mutation.
The European Medicines Agency has issued a positive opinion for the antisense oligonucleotide medicine Spinraza (nusinersen) to treat patients with spinal muscular atrophy, a rare genetic disease that causes muscle weakness and a progressive loss of movement.
Vernalis Plc has had its application for the cough-cold medicine CCP-07 turned back by the US Food and Drug Administration, with the result that the product will not reach the US market as quickly as planned.
The US Food and Drug Administration has turned back an application from Eli Lilly and Company for baricitinib, a candidate treatment for rheumatoid arthritis, saying that more data is needed to determine the most appropriate doses of the drug for use in patients.
The European Medicines Agency EMA) has formalised its ongoing relationship with universities in order to tap into research that could lead to new medicines and provide regulatory guidance to academic entrepreneurs.