Prescription Drug User Fee Act (PDUFA) deadline extended by three months for further submission review until September 25, 2021
COPENHAGEN, Denmark, June 11, 2021 (GLOBE NEWSWIRE) – Ascendis Pharma A / S (Nasdaq: ASND), a biopharmaceutical company that uses its innovative TransCon technologies to create new product candidates that address unmet medical needs, announced today that the United States Food and Drug Administration (FDA) informed the Company that the information submitted by the Company as part of the ongoing FDA review of the Biologics License Application (BLA) for lonapegsomatropin for the treatment of pediatric growth hormone deficiency (GHD) was a major amendment to BLA. As a result, the FDA has extended the deadline for the Prescription Drug User Fee Act by three months, until September 25, 2021.
“We have answered all outstanding FDA questions and believe that the full package we have submitted meets all of the FDA’s requests and will allow for a full review of the application of lonapegsomatropin for pediatric GHD,” said said Jan Mikkelsen, President and CEO of Ascendis Pharma. Officer. “We are committed to making lonapegsomatropin the market leader in the treatment of pediatric GHD and look forward to continued interactions with the FDA during the remainder of the review process. “
About Ascendis Pharma A / S
Ascendis Pharma applies its innovative platform technology to create a leading fully integrated biopharmaceutical company focused on improving the lives of patients. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new therapies potentially best in class.
Ascendis Pharma currently has a pipeline of three independent endocrinology product candidates for rare diseases and one oncology product candidate under clinical development. The company continues to expand into other therapeutic areas to address unmet patient needs.
Ascendis is headquartered in Copenhagen, Denmark, with additional facilities in Heidelberg and Berlin, Germany, Palo Alto and Redwood City, California, and Princeton, New Jersey.
Please visit www.ascendispharma.com (for worldwide information) or www.ascendispharma.us (for US information).
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical fact, included in this press release regarding the future operations, plans and objectives of the management of Ascendis are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ ability to apply its platform technology to build a leading and fully integrated biopharmaceutical company, (ii) the portfolio of Ascendis ‘products and its expansion into additional therapeutic areas, (iii) Ascendis’ expectations regarding its ability to use its TransCon technologies to create potentially best-in-class new therapies, (iv) the PDUFA date for the BLA of lonapegsomatropin for the treatment of pediatric GHD, (v) Ascendis ‘expectations regarding the potential for approval of lonapegsomatropin as a treatment for pediatric GHD and (vi) Ascendis’ ability to produce lonapegsomatropin the market leading treatment for the treatment of pediatric GHD. Ascendis may not carry out the plans, realize the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on such forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from Ascendis’ forward-looking statements, including the following: unanticipated safety or efficacy results in its oncology programs, lonapegsomatropin, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the development and potential commercialization of its oncology programs, lonapegsomatropin, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and the activities of Ascendis in general; delays in the development of its oncology, lonapegsomatropin, TransCon PTH and TransCon CNP programs or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; reliance on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to secure additional funding, if necessary, to support its business operations and the effects on its business of the global COVID-19 pandemic. For a more detailed description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as the risks associated with Ascendis ‘business in general, see Ascendis’ annual report on Form 20- F filed with the United States Securities and Exchange Commission (SEC) on March 10, 2021 and other future Ascendis reports filed or submitted to the SEC. Forward-looking statements do not reflect the potential impact of any future licenses, collaborations, acquisitions, mergers, disposals, joint ventures or investments that Ascendis may enter into or make. Ascendis assumes no obligation to update forward-looking statements, except as required by law.
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