The European Medicines Agency is recommending approval of Natpar (parathyroid hormone) to treat patients with chronic hypoparathyroidism who require more than calcium and vitamin D. If approved by the European Commission, Natpar would be the first hormone replacement therapy for this condition.
The US Food and Drug Administration has approved the monoclonal antibody brodalumab to treat adults with moderate-to-severe plaque psoriasis, an autoimmune disorder that causes patches of redness and flaking on the skin.
The coming into force next year of new European rules governing clinical trials will simplify trial application procedures and make more study information available to the public. At a meeting of the European Forum for Good Clinical Practice on 22 February, healthcare professionals discussed how the new rules on transparency should build public confidence in the regulatory system and avoid potentially serious accidents.
The Food and Drug Administration has approved a second drug for the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes a progressive deterioration of muscle. The treatment is a corticosteroid called Emflaza (deflazacort) that works by decreasing inflammation and reducing the activity of the immune system.
With the UK government on course to begin the legal process for leaving the EU, industry leaders have spoken openly about the impact. In separate teleconferences, the CEOs of AstraZeneca Plc and GlaxoSmithKline Plc (GSK) said the EMA would almost certainly have to re-locate.
Ingmar Hoerr, chief executive of CureVac AG, has been appointed as one of 15 members of a new group that will advise the European Commission on strengthening the environment for research and innovation in the European Union.
A proposed liberalisation of rules that govern financial transactions in the European Union is the subject of a public consultation that started on 20 January and will continue until 17 March. The consultation is being managed by the European Commission.
The Food and Drug Administration (FDA) has given its first approval to a drug for spinal muscular atrophy, a hereditary disease that causes weakness and muscle wasting. The drug, Spinraza (nusinersen), is an antisense oligonucleotide that binds to RNA in order to regulate gene expression.
A new JAK inhibitor for patients with rheumatoid arthritis (RA) has been recommended for approval by the European Medicines Agency. If authorised by the European Commission, it would be the first RA drug to be launched in Europe with this mechanism of action.
The UK is to allow a technique for preventing an inherited disorder known as mitochondrial disease in cases where other treatments are ineffective. The treatment, known as mitochondrial donation, was approved on 15 December.