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February 22, 2021

Investigator-sponsored Phase II/III for Pfizer’s sirolimus for Sturge-Weber Syndrome sees 15-point surge in FDA regulatory potential

As of 15 February, the likelihood of approval (LoA) for Pfizer’s Rapamune (sirolimus) for Sturge-Weber Syndrome (SWS) rose 15 points, according to GlobalData’s LoA data.

By William Newton

As of 15 February, the likelihood of approval (LoA) for Pfizer’s Rapamune (sirolimus) for Sturge-Weber Syndrome (SWS) rose 15 points, according to GlobalData’s LoA data. The score was based on published results from an investigator-sponsored Phase II/III trial that saw sirolimus achieve a statistically significant improvement in one aspect of the primary endpoint.

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The 10-patient Phase II/III) used change in cognitive functioning after six months as its primary outcome. The results were assessed using a panel of testing developed by SWS experts that included measures from the National Institute of Health Toolbox, according to ClinicalTrials.gov. The published data reported a statistically significant improvement in the Pattern Comparison Processing Speed Test, but did not show statistical significance in any other assessments in the primary endpoint, including in measures of executive function, attention, episodic memory, language skills, working memory, or patient-reported emotional functioning (Sebold A.J., et al. (2020) Paediatric Neurology 115: 29-40). Of the ten patients enrolled, one had a moderate upper respiratory infection possibly related to the drug while all other reported adverse events were mild.

While the LoA prior to this news was 8%, GlobalData’s analysis using a combination of machine learning and a proprietary algorithm has raised the LoA to 23%. Rapamune is currently marketed for kidney transplant rejection, angiofibroma and lymphangioleiomyomatosis.

William Newton is a Reporter for Clinical Trials Arena parent company GlobalData’s investigative journalism team. A version of this article originally appeared on the Insights module of GlobalData’s Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.

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How attractive are current investment opportunities in Europe?

Europe has been identified as one of the most favorable regions for investors, seeing high investment activity in the past year. Most of these investments have been through Debt Offering, valued at close to $700 billion. The region has provided attractive investments in a diverse set of companies. Companies who tend to major themes such Digital Media, Cloud, Artificial Intelligence, E-commerce, and Big Data are recording the highest number of deals, with Digital Media recording close to 2,000 deals. However, GlobalData’s whitepaper offers a full view of the market, analyzing less successful or attractive points of investment as well, examining statistics on Equity Offering investments and PE/VC deals. Understand how government agencies for economies around the world use GlobalData Explorer to:  
  • Track the M&A and Capital Raising volumes into their target market
  • Identify the top sectors in the target market attracting the investments
  • For any investment segment, identify the top Investors inside and outside the target economy that are already investing in the Segment
  • Assess and showcase the growth potential for various Industries in the target economy
Don’t miss out on key market insights that can help optimize your next investment – read the report now.
by GlobalData
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