rare diseases
Shire’s drug picked up from Baxalta given fast track designation
March 23, 2017Research and Development, Sales and Marketing Shire, rare diseases
Shire has announced that its drug, SHP655, has received a fast track designation based on pre-clinical trials and positive results …
Horizon and Fulcrum Therapeutics form CRISPR collaboration
September 6, 2016Manufacturing and Production, Research and Development Horizon Discovery, crispr, fulcrum, fulcrum therapeutics, gene editing, horizon, rare diseases
Horizon Discovery has announced that it has entered into a partnership with Fulcrum Therapeutics to utilise Horizon’s CRISPR-based screening platform …
Drug repurposing can help tackle Rare Diseases, says charity
February 29, 2016Medical Communications, Research and Development, Sales and Marketing rare diseases
On Rare Disease Day 2016, UK charity Findacure says existing drugs may hold the key to new treatments and repurposing …
More evidence required for Alexion’s Kanuma, says NICE
February 18, 2016Medical Communications, Research and Development Alexion, Kanuma, LAL-D, rare diseases
NICE says it needs additional trial data before it can recommend Alexion’s enzyme replacement therapy Kanuma (sebelipase alfa) for the …
Novartis forms Mereo as UK biotech spin-off
July 29, 2015Research and Development Mereo, Novartis, biotech, rare diseases
Novartis has formed a new UK biotech company by selling three investigational drugs in exchange for shares in the new …
Overcoming barriers in rare diseases
June 22, 2015Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing BIA, NHS, Shire, bates, government, nps, rare diseases
In recent months the focus on the need to develop new drugs and fund new treatments for rare diseases has …
Government lays out rare diseases plan
March 5, 2015Sales and Marketing Department of Health, NHS, UK, government, howe, rare diseases
The UK government has set out new plans to improve the lives of patients with rare diseases by 2020. The …
Pfizer signs universities deal
May 12, 2014Research and Development, Sales and Marketing AstraZeneca, Pfizer, academics, rare diseases
Pfizer has signed a five-year research deal with several top British universities to hunt for new treatments for rare disease. …
RPS: ‘Give pharma incentives’
May 8, 2014Research and Development, Sales and Marketing NHS, RPS, rare diseases, royal pharmaceutical society
Incentives are urgently required to speed up the development of personalised medicines, according to a new report from the Royal …
EU regulator recommended 81 medicines in 2013
January 21, 2014CHMP, EC, EU, FDA, US, rare diseases
Last year the Committee for Medicinal Products for Human Use (CHMP) recommended 81 medicines for marketing approval, compared with just …
Rare disease fund scrapped by NHS England
October 16, 2013Research and Development, Sales and Marketing NHS England, fund, rare diseases
NHS England has pulled the plug on a £50 million fund to advance innovation in the investigation and treatment of …
Rare disease drugs ‘need separate budget’
September 3, 2013Research and Development, Sales and Marketing NHS, NICE, rare diseases
A group of MPs in the UK are calling for a ring-fenced fund to pay for drugs for rare and …
Chiesi snaps up Zymenex
August 29, 2013Sales and Marketing Chiesi, lamazym, rare diseases, zymenex
Chiesi Group is to acquire biopharma company Zymenex Holdings, allowing the Italian firm to enter the rare disease arena for …
Scotland fund for rare diseases
January 17, 2013Sales and Marketing SMC, Scotland, rare diseases
The Scottish government is launching a £21 million fund in March to meet the costs of patients who require orphan …
Rare diseases – pharma’s new frontier
March 26, 2012Business Services, Manufacturing and Production, Medical Communications, Research and Development, Sales and Marketing Big Pharma, CAPS, McConaghie, rare diseases
The last ten years has seen our understanding of rare diseases move ahead rapidly, often thanks to greater understanding of …
