47 documents

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22:53 , Jun 28, 2019 |  BC Extra  |  Clinical News

Pfizer planning Phase III for DMD gene therapy, close on Sarepta's heels

Undeterred by two patient hospitalizations revealed in Phase Ib data Friday for its DMD gene therapy, Pfizer is moving forward with plans for a Phase III trial start in 1H20, setting up a tight race...
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21:56 , Jun 28, 2019 |  BC Extra  |  Clinical News

June 28 Clinical Quick Takes: Imfinzi extends survival in Phase III SCLC study; plus LSK, Pfizer, FibroGen, Aclaris and more

AZ's Imfinzi shows survival benefit in Phase III for SCLC  AstraZeneca plc (LSE:AZN; NYSE:AZN) said an interim analysis of the Phase III CASPIAN trial showed Imfinzi plus etoposide and platinum-based chemotherapy met the primary endpoint...
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22:58 , Dec 20, 2018 |  BC Extra  |  Politics & Policy

FDA releases next guidance in patient-focused drug development series

FDA released draft guidance on Thursday to encourage industry and other stakeholders to submit proposed draft guidances for incorporating patient experience data into product development and regulatory decisions. The agency said stakeholders could submit their...
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23:58 , Jun 21, 2018 |  BC Extra  |  Company News

Sarepta says no to right to try for its DMD therapies

Following data from a small trial of a Duchenne muscular dystrophy gene therapy, Sarepta Therapeutics Inc. (NASDAQ:SRPT) told BioCentury that it will not offer pre-approval access under right-to-try legislation or FDA’s expanded access program for...
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00:54 , Feb 24, 2018 |  BioCentury  |  Regulation

Herculean task for DMD

Driven by the delayed reimbursement decision for Europe’s only approved DMD therapy, the patient advocacy group Duchenne UK has brought together seven companies in the space to create a set of shared tools that should...
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22:30 , Feb 2, 2018 |  BioCentury  |  Product Development

AA Viral meme

Gene therapy pioneer James Wilson’s revelation of two new types of toxicity in animals receiving high systemic doses of adeno-associated viral vectors is unlikely to herald a major disruption of the AAV space, though it...
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23:55 , Jan 4, 2018 |  BC Extra  |  Financial News

SomaLogic completes $200M financing round

Protein analysis company SomaLogic Inc. (Boulder, Colo.) said it completed a $200 million tranched financing round led by iCarbonX. Nan Fung Life Sciences and Madryn Asset Management also participated. SomaLogic's SOMAscan technology can measure 5,000...
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01:29 , Dec 23, 2017 |  BioCentury  |  Regulation

Prioritizing preferences

The realization that patients have distinct preferences about the trade-offs and risks inherent in medical decisions -- and that their choices are frequently different from those physicians or regulators would make -- has mobilized FDA,...
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20:22 , Nov 3, 2017 |  BioCentury  |  Regulation

Frame work

A little over four years into its implementation, FDA’s structured benefit-risk framework appears to be meeting its objectives of creating transparency into its approval decisions. The agency began to incorporate the framework templates into its...
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19:11 , Oct 13, 2017 |  BioCentury  |  Regulation

Seeking surrogates

Thanks to flaws in PTC Therapeutics Inc. ’s trials, the recent FDA advisory committee meeting to discuss Translarna ataluren did nothing to clarify what level of dystrophin expression would be necessary to support an accelerated...
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