The week in industry: US FDA awards 12 grants to fund rare disease treatment programs

This week: US$3 million payment for nomination of new GPCR target, orphan drug designation granted for soft tissue sarcoma treatment and erythropoietic protoporphyria drug approved for marketing in the US.

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Oct 10, 2019
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This week's industry news:  

US FDA awards 12 grants to fund rare disease treatment programs
$3 million payment for nomination of new GPCR target
Orphan drug designation for soft tissue sarcoma treatment
Erythropoietic protoporphyria drug approved for marketing in the US


US FDA awards 12 grants to fund rare disease treatment programs

As part of the Orphan Products Clinical Trial Grants Program, the US FDA will be awarding 12 new clinical trial research grants totaling more than US$15 million over the course of the next 4 years. The grants are focused towards programs that are working to meet the needs of patients impacted by a variety of rare diseases.

“For more than 35 years, the FDA has been providing much-needed financial support for clinical trials of potentially life-changing treatments for patients with rare diseases. To date, the Orphan Products Clinical Trials Grants Program’s grants have supported research that led to the marketing approval of more than 60 treatments for rare diseases,” commented FDA Principal Deputy Commissioner Amy Abernethy. “We are encouraged by the amount of interest we continue to have in the grants program and are committed to working with researchers and industry to facilitate and support the study and development of treatments for patients with rare diseases.”

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$3 million payment for nomination of new GPCR target

Following on from the initial license agreement in July 2019, Genentech (CA, USA) has notified Sosei Heptares (Tokyo, Japan) of their desire to nominate a new G protein-coupled receptor (GPCR) disease target. The nominated target is part of the promising nature of the collaboration and Genentech will be responsible for developing any new drugs for this target. Sosei have received an upfront payment of US$3 million that has the potential to exceed US$1 billion for future milestones.

“We are delighted that our collaboration with Genentech has rapidly nominated an additional GPCR disease target. This is a great example of how we can leverage our world leading GPCR expertise to quickly develop productive working relationships with our partners to deliver on key project milestones. We look forward to advancing this important collaboration as we capitalize on our complementary expertise to generate potential new drug candidates to GPCR disease targets,” commented Malcolm Weir, Executive Vice President & Chief R&D Officer at Sosei.

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Orphan drug designation for soft tissue sarcoma treatment

The European Commission has granted nirogacestat – an oral, small molecule, γ-secretase inhibitor for the treatment of soft-tissue sarcoma – orphan drug designation. The drug is currently in Phase III clinical trials (DeFi trial), in adults with progression desmoid tumors – a type of soft-tissue tumor, for which there are currently no approved treatment options.

“This Orphan Drug Designation in the European Union is another important development for SpringWorks and follows the Orphan Drug, Fast Track and Breakthrough Therapy Designations already granted for nirogacestat in the U.S. by the FDA,” commented Saqib Islam, Chief Executive Officer of SpringWorks, who developed the therapy. “We are currently enrolling adult patients in our DeFi trial and will continue to work closely with global regulators with the goal of bringing nirogacestat to patients as quickly as possible.”

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Erythropoietic protoporphyria drug approved for marketing in the US

The US FDA granted marketing approval of SCENESSE®, the first and only systemic photoprotective drug for the treatment of erythropoietic protoporphyria (EPP) – a rare metabolism disorder. The drug, developed by Clinuvel Pharmaceuticals (Melbourne, Australia), is indicated to treat EPP in adults by increasing pain free light exposure.

“This event is transformational in that we are now accelerating our exchange with the FDA and European Medicines Agency to expand the use of Scenesse in additional indications, as the passing of time has unveiled that our breakthrough technology deserves wider application in modern medicine. I am confident that our teams will understand going forward that art and skills will be required to execute against due dates while containing the costs. If the past had been arduous, the hard labor is just about to start,” commented Philippe Wolgen, CEO of Clinuvel.

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