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Orchard Therapeutics’ war chest balloons with $200M IPO fiercebiotech
November 02, 2018
Orchard Therapeutics priced its IPO at $14 a share, raising $200 million and exceeding the $173 million goal it set earlier this month. The funds will bankroll the pivotal trials and...
Novartis, Pfizer join forces on potentially lucrative fatty liver disease expressbpd
October 30, 2018
The collaboration will help develop combination therapies involving medicines they have been working on separately to treat non-alcoholic steatohepatitis, or NASH
Alnylam eyes quickie givosiran filing after hitting interim goal fiercebiotech
September 28, 2018
Alnylam is set to file for accelerated FDA approval of givosiran on the strength of interim data from a phase 3 trial.
Alexion snaps up another biotech, paying $1.2B for Syntimmune fiercebiotech
September 28, 2018
Alexion’s rebuilding plan has continued with another bolt-on acquisition, this time a $400 million upfront deal for rare disease specialist Syntimmune.
FDA Awards 12 Grants to Fund New Clinical Trials americanpharmaceuticalreview
September 26, 2018
The U.S. Food and Drug Administration (FDA) has awarded 12 new clinical trial research grants totaling more than $18 million over the next four years to enhance the development of medical products for patients with rare diseases.
Supernus adds epilepsy candidate with $185M Biscayne buy fiercebiotech
September 18, 2018
Supernus' drug will be developed for rare childhood epilepsy disorder Dravet syndrome.
Pfizer eyes ATTR cardiomyopathy filing for tafamidis after acing phase 3 trial fiercebiotech
August 28, 2018
Analysts suggest the drug could reach the market in 2019
Healx reels in $10M in series A funding for work on rare diseases fiercebiotech
July 31, 2018
Healx, a U.K.-based company that works with patient advocacy groups to repurpose drugs for rare diseases, reeled in $10 million in series A funding it plans to use to hire more researchers and enhance its AI platform.
New Global Commission to accelerate diagnostic time for children with rare diseases biospectrumasia
March 05, 2018
Alliance aims to shorten the often multi-year journey that patients and families endure before being diagnosed with a rare disease
China: Shortage of doctors and medicine for rare diseases leads to more overseas purchase en-cphi.cn
October 25, 2017
36 specific reform measures has been proposed recently, including policies to encourage pharmaceutical products and medical devices for rare diseases, such as conditional approval for marketing...

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