The FDA has granted Amicus’ AT-GAA a Breakthrough Therapy Designation (BTD) for the treatment of late onset Pompe disease.
News
Novartis to licence Akcea’s lipoprotein lowering drug
Novartis has announced that it is exercising its option to license the rights to develop and commercialise Akcea’s TQJ230.
Brilinta hits targets in PhIII cardiovascular/diabetes trial
AstraZeneca’s Brilinta has been found to reduce cardiovascular events in patients with coronary heart disease and diabetes but no prior heart attack or stroke.
Boehringer Ingelheim appoints new chief medical officer
Boehringer Ingelheim has announced the appointment of Dr Mehdi Shahid as chief medical officer.
Santhera’s Syros succesful in DMD study
Santhera Pharmaceuticals has announced results from its Syros DMD study.
Ipsen acquires Clementia in bid to boost rare disease portfolio
Ipsen has announced plans to acquire Clementia Pharmaceuticals.
Roche to pay $4.3bn for Spark in gene therapy move
Roche has announced plans to buy Spark Therapeutics for $4.3 billion.
Research finds PTSD affects 8% of under 18s
One in 13 under 18 year-olds has been found to have post-traumatic stress disorder (PTSD) in a new study.
Cancer screening programmes to get an independent review
A major cancer screening overhaul has been launched as part of the NHS Long Term Plan’s renewed drive to improve care and save lives.
Boehringer Ingelheim signs drug discovery pact with 3B Pharmaceuticals
3B Pharmaceuticals has announced a multi-year collaboration with Boehringer Ingelheim, for peptide-led drug discovery.
Prescriptions to rise to £9 from April
The government has announced another rise in prescription costs for medicines dispensed on the NHS in England, from £8.80 to £9 as of April 1 this year.
FDA hits leukemia study with partial clinical hold
The FDA has placed a partial clinical hold on Xencor’s Phase I study of XmAb14045 on safety concerns.
Morphic and Janssen team up for integrin therapies
Morphic and Janssen have entered into a research and development collaboration to discover and develop novel integrin therapeutics.
Pfizer adjusts Xeljanz trial due to pulmonary embolism risk
Pfizer has announced plans to lower its Xeljanz (tofacitinib) trial dosing from 10mg twice-daily to 5mg twice-daily, after pulmonary embolisms were found to be more frequent among patients treated with the higher dose.
Brineura hit with NICE rejection
NICE is rejecting NHS funding for a £500,000-a-year drug to treat Batten disease on concerns over its long-term effectiveness.
