Fibrodysplasia ossificans progressiva

Retinoic Acid Receptor Agonists Drug Pipeline Research Report 2022 - ResearchAndMarkets.com

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Monday, August 14, 2023
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The "Retinoic acid receptor agonists - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Retinoic acid receptor agonists - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The report serves as an exhaustive source of insights into the landscape of Retinoic Acid Receptor (RARs) Agonist pipeline, encompassing over 15 companies and 15 pipeline drugs.
  • The ongoing development of therapies revolves around innovative strategies, aiming to address the intricacies of Retinoic Acid Receptor (RARs) Agonist.
  • Retinoic Acid Receptor (RARs) Agonist: Therapeutic Assessment
    This segment of the report provides insights about the different Retinoic Acid Receptor (RARs) Agonist drugs segregated based on following parameters that define the scope of the report.

Ipsen delivers solid H1 2023 results and upgrades its full-year guidance

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Thursday, July 27, 2023
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The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.

Key Points: 
  • The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.
  • The Company’s auditors performed a limited review of the H1 2023 condensed consolidated financial statements.
  • Ipsen intends to publish its year-to-date and third-quarter sales update on 26 October 2023.
  • The performance shown in this announcement covers the six-month period to 30 June 2023 (H1 2023) and the three-month period to 30 June 2023 (Q2 2023), compared to six-month period to 30 June 2022 (H1 2022) and the three-month period to 30 June 2022 (Q2 2022), respectively, unless stated otherwise.

Ipsen announces European Commission decision on palovarotene for the treatment of FOP

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Wednesday, July 19, 2023
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Palovarotene is the first treatment to be submitted anywhere in the world for regulatory approval for FOP, an ultra-rare disease with approximately 900 known cases worldwide.

Key Points: 
  • Palovarotene is the first treatment to be submitted anywhere in the world for regulatory approval for FOP, an ultra-rare disease with approximately 900 known cases worldwide.
  • “We worked tirelessly to bring a greatly needed treatment option to patients living with FOP in the E.U.,” said Howard Mayer, Executive Vice President and Head of Research and Development for Ipsen.
  • We are therefore disappointed that the European Commission decided not to approve this treatment for patients with FOP in Europe.
  • This included MOVE, the first and largest Phase III clinical trial for FOP, a condition that causes abnormal bone growth.

Ipsen announces positive outcome of FDA Advisory Committee on investigational palovarotene for fibrodysplasia ossificans progressiva

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Thursday, June 29, 2023
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The FDA is currently reviewing the New Drug Application (NDA) for palovarotene with a decision anticipated by August 16, 2023.

Key Points: 
  • The FDA is currently reviewing the New Drug Application (NDA) for palovarotene with a decision anticipated by August 16, 2023.
  • If approved, palovarotene will be the first treatment in the U.S. for FOP.
  • “We are pleased with the outcome today and believe that the vote of the FDA advisory committee conveys the potential of palovarotene in helping manage the severe impacts of FOP.
  • Palovarotene is currently authorized for use in appropriate patients in Canada and United Arab Emirates where it is marketed as SohonosTM (palovarotene capsules).6

Ipsen receives CHMP negative opinion, following re-examination of potential first FOP treatment in the E.U.

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Friday, May 26, 2023
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Palovarotene is the first medicine to be submitted for regulatory approval for FOP.

Key Points: 
  • Palovarotene is the first medicine to be submitted for regulatory approval for FOP.
  • are for managing the symptoms caused by FOP, such as inflammation and chronic pain and not the underlying disease.
  • and for the clinicians managing this chronic and progressive disease,” said Dr. Genevieve Baujat, Clinical Geneticist Consultant at Necker-Enfants Malades Hospital, Paris, France.
  • “Many of my colleagues and I have been waiting for a long time for a treatment that can help us manage the devastating disease that is FOP.

Ipsen receives new FDA PDUFA date for investigational palovarotene for the treatment of people with FOP

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Thursday, March 16, 2023
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Additional information on palovarotene clinical trial data, requested in a complete response letter to Ipsen in December 2022, will be reviewed as part of this resubmission process.

Key Points: 
  • Additional information on palovarotene clinical trial data, requested in a complete response letter to Ipsen in December 2022, will be reviewed as part of this resubmission process.
  • Furthermore, Ipsen has requested a re-examination of the European Medicines Agency’s January 2023 Committee for Medicinal Products for Human Use (CHMP) opinion on palovarotene.
  • Palovarotene is authorized for use in appropriate patients in Canada where it is marketed as SohonosTM (palovarotene capsules).
  • Investigational palovarotene is under review with several regulatory authorities.

Ipsen Receives CHMP Negative Opinion for Palovarotene as a Treatment for Fibrodysplasia Ossificans Progressiva in E.U.

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Friday, January 27, 2023
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Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP).

Key Points: 
  • Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not to grant marketing authorization for investigational palovarotene as a treatment for the ultra-rare bone disease, fibrodysplasia ossificans progressiva (FOP).
  • there are currently only symptomatic treatments for FOP, which do not reduce the formation of extra-skeletal bone in patients with the condition.
  • Ipsen will be requesting a re-examination of the CHMP opinion, based on scientific data available from the existing palovarotene clinical trial program.
  • We in the FOP community have been waiting a long time for innovations to treat this disabling disease.”

Ipsen receives Complete Response Letter for palovarotene, an investigational treatment for fibrodysplasia ossificans progressiva

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Friday, December 23, 2022
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The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation (heterotopic ossification) in people living with fibrodysplasia ossificans progressiva (FOP).

Key Points: 
  • The U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL) regarding the New Drug Application for palovarotene, an investigational treatment for the reduction of new abnormal bone formation (heterotopic ossification) in people living with fibrodysplasia ossificans progressiva (FOP).
  • The CRL is related to the regulatory agency’s previous request for additional information on palovarotene clinical trial data communicated to Ipsen in October 2022, which is not a request for additional efficacy or safety data beyond existing studies.
  • Ipsen anticipates responding to the request in the first quarter of 2023 with an expected six-month FDA review cycle.
  • The FDA has not announced a rescheduled date for the Endocrinologic and Metabolic Drugs Advisory Committee meeting for investigational palovarotene.

Ipsen Statement on Updated Timeline for Palovarotene FDA Advisory Committee Meeting

Retrieved on: 
Tuesday, October 25, 2022
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Ipsen (Euronext: IPN; ADR: IPSEY) reports that the U.S. Food and Drug Administration (FDA) has today announced its decision to postpone the planned Endocrinologic and Metabolic Drugs Advisory Committee meeting for investigational palovarotene until a later date to be confirmed.

Key Points: 
  • Ipsen (Euronext: IPN; ADR: IPSEY) reports that the U.S. Food and Drug Administration (FDA) has today announced its decision to postpone the planned Endocrinologic and Metabolic Drugs Advisory Committee meeting for investigational palovarotene until a later date to be confirmed.
  • The original advisory committee meeting was scheduled for 31 October 2022.
  • The FDA informed Ipsen that the postponement relates to an FDA request for new information on palovarotene clinical trial data and does not relate to the safety profile of palovarotene.
  • We will work expediently with the FDA as we remain committed to bringing innovative treatment options to the Fibrodysplasia Ossificans Progressiva community.

FDA Grants Orphan Drug Designation for BioCryst’s ALK-2 Inhibitor, BCX9250, for the Treatment of Fibrodysplasia Ossificans Progressiva

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Wednesday, August 31, 2022
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RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BCX9250 for the treatment of fibrodysplasia ossificans progressiva (FOP).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BCX9250 for the treatment of fibrodysplasia ossificans progressiva (FOP).
  • We appreciate the FDAs decision to grant orphan drug designation to BCX9250 as we work toward our goal of bringing this important oral investigational therapy to FOP patients.
  • Earlier this year, BioCryst announced that BCX9250 has received Fast Track designation from the FDA, in addition to orphan drug designation and PRIME designation from the European Medicines Agency (EMA).
  • This press release contains forward-looking statements, including statements regarding BioCrysts plans and expectations for BCX9250 and the potential benefits associated with an FDA orphan drug designation.