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FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases firstwordpharma
March 23, 2019
One key way we’ve addressed these opportunities is by providing funding for rare disease natural history ……
Pharmaceutical Collaborations and Rare Diseases Drive the Drugs to Watch as New Blockbusters Are Forecast to Hit the Market in 2019 en-cphi.cn
March 21, 2019
Clarivate Analytics announced today the launch of its annual "Cortellis Drugs to Watch" report.
UK prioritises rare diseases in genomic medicine strategy pharmaceutical-technology
February 28, 2019
In preparation for Rare Disease Day on 28 March, the UK Government has announced a National Genomic Healthcare Strategy that specifically focuses on providing predictive, personalised health services to people with rare diseases....
As promised, Novartis eyes new method for funding ultrapricey next-gen drugs: FT fiercepharma
December 19, 2018
Novartis is weighing an insurance method often associated with natural disasters to help win coverage for its pricey next-generation cell and gene therapies.
U.K. cost watchdog NICE issues preliminary ‘no’ on Alnylam’s Onpattro and Ionis’ Tegsedi fiercepharma
December 14, 2018
Alnylam and Ionis are in a head-to-head battle for market share with their new treatments for the rare disease hereditary transthyretin-mediated amyloidosis (ATTR)...
Takeda CEO gets his Shire megamerger, thanks to shareholder nod. Can he make it pay off? fiercepharma
December 06, 2018
Behold, Takeda can soon call itself a Big Pharma. Its shareholders joined Shire’s in separately approving the mega-M&A deal, despite opposition from a small group of dissidents...
Catalyst's long-delayed Firdapse snags FDA nod, but pricing questions loom fiercepharma
December 05, 2018
It took a breakthrough designation, orphan drug status, an embarrassing up-front rejection letter and some extra trials, but Catalyst Pharmaceuticals has finally crossed the finish line with Firdapse.
ASH: With Gamifant launch rolling, Sobi unveils more data backing the rare disease drug fiercepharma
December 05, 2018
Armed with last month's FDA approval, Sobi is rolling out its newly minted rare disease drug Gamifant with a small, targeted sales force, and Monday it unveiled more of the data that won the agency’s favor in the first place.
FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases fda
November 09, 2018
The U.S. Food and Drug Administration today announced that it has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to enhance
ProQR licenses Ionis’ RNA drug for inherited blindness fiercebiotech
November 02, 2018
ProQR is licensing an RNA medicine from Ionis Pharmaceuticals for the treatment of retinitis pigmentosa, a form of rare, inherited blindness. The plan is to start a phase 1/2 trial of...

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