Business Wire

Ipsen Announces U.S. FDA Priority Review for palovarotene New Drug Application in Patients With Fibrodysplasia Ossificans Progressiva Following Resubmission

Share

Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review its resubmitted New Drug Application (NDA) for investigational palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disorder. Ipsen is seeking approval of palovarotene, an oral, selective retinoic-acid receptor gamma (RARγ) agonist for the prevention of heterotopic ossification (HO; new bone formation outside of the normal skeletal system). The FDA has assigned 29 December 2022 as the Prescription Drug User Fee Act goal date, which is on track with anticipated regulatory submission timelines.

“This FDA submission acceptance marks a significant milestone for those living with FOP in the U.S. where no approved treatment options exist today for this progressive and debilitating ultra-rare genetic disorder,” said Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen. “Ipsen is committed to developing life-changing treatments for patients living with rare diseases despite the challenges that exist when there are few, or no precedents to build on when developing a new, innovative medicine for a complex condition. We remain committed to the FOP community, and we hope the Priority Review is one step closer towards getting a much needed treatment to people living with FOP as soon as possible.”

The FDA’s Priority Review designation is assigned to investigational therapies that, if approved, represent a significant improvement in the safety or effectiveness of the treatment compared to the current standard of care.

In January 2022, Health Canada was the first regulatory authority to approve SohonosTM (palovarotene capsules) to reduce the formation of HO in adults and children aged 8 years and above for females and 10 years and above for males with FOP. Sohonos is approved in Canada for the treatment of patients with FOP for both chronic use and for flare-ups in these patient populations. Palovarotene is not currently approved outside of Canada. A Marketing Authorization Application for palovarotene has also been filed in the E.U. in 2021, with responses to questions raised by the European Medicines Agency submitted in June 2022, in line with the ongoing regulatory process. Ipsen anticipates additional applications to other regulatory agencies in due course.

FOP has an estimated prevalence of 1.36 per million individuals; however, the number of confirmed cases varies by country.1,2 It is characterized by new bone formation outside of the normal skeletal system, like in soft connective tissues, a process known as HO,[3] which can be preceded by painful soft-tissue swelling or ’flare-ups’.2 Flare-up episodes are common and are a substantial contributor to the formation of new HO, however HO can form in the absence of a flare-up. HO, once formed, is irreversible and leads to loss of mobility and shortened life expectancy.3

# # #

About palovarotene
Palovarotene is an oral investigational, selective retinoic-acid receptor gamma (RARγ) agonist being developed as a potential treatment for people living with the debilitating ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP). Palovarotene, which received rare pediatric disease and Breakthrough Therapy Designations from the U.S. Food and Drug Administration (FDA) for the potential treatment of FOP, was acquired by Ipsen through the acquisition of Clementia Pharmaceuticals in April 2019. The palovarotene New Drug Application (NDA) was initially accepted by the FDA for Priority Review on May 28, 2021, and Ipsen announced withdrawal of the NDA on August 13, 2021. The resubmission of the palovarotene NDA has been accepted by the FDA for Priority Review in the U.S.

About fibrodysplasia ossificans progressiva (FOP)
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by bone that forms outside the normal skeleton, in muscles, tendons, or soft tissue.3 FOP has an estimated prevalence of 1.36 per million individuals globally; however, the number of confirmed cases varies by country.1,2 The median age at time of FOP diagnosis is five years old.4

About Ipsen
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With Specialty Care sales of €2.6bn in FY 2021, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen, excluding its Consumer HealthCare business, has around 4,500 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information visit www.ipsen.com.

Ipsen’s forward looking statements
The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s 2021 Universal Registration Document, available onipsen.com.

1 Lilijesthrom, M & Bogard, B. ‘The global known FOP population’, FOP Drug Development Forum, Boston, MA, 24-25 October 2016.
2 Baujat, G et al. Prevalence of fibrodysplasia ossificans progressiva (FOP) in France: an estimate based on a record linkage of two national databases. Orphanet Journal of Rare Diseases. 2017; 12:123.
3 Kaplan, FS, et al. The medical management of fibrodysplasia ossificans progressiva: current treatment considerations. Proc Intl Clin Council FOP. 2019; 1:1-111.
4. Pignolo, RJ et al. Bone. 2020; 134:115274.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

For further information:

Investors
Craig Marks
Vice President, Investor Relations
+44 7584 349 193

Adrien Dupin de Saint-Cyr
Investor Relations Manager
+33 6 64 26 17 49

Media
Maryann Quinn
Senior Director, US Communications
+1 857 529 1151

Anna Gibbins
Global Head Franchise Communications, Rare Diseases
+44 7717 801900

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

IFF Unveils Nourish Innovation Lab in Union Beach, New Jersey19.8.2022 12:55:00 CEST | Press release

IFF (NYSE:IFF) today announced the opening of its new Nourish Innovation Lab at the company’s Union Beach, New Jersey research and development center. In the state-of-the-art lab, scientists—including flavorists and food designers—will leverage tools, technologies and ingredient expertise to offer customers full product design, driving growth in the global food and beverage markets by fast forwarding innovation. “This new facility is a testament to how we pioneer innovation and drive speed to market and accessibility to our customers,” said Nicolas Mirzayantz, president of Nourish, IFF. “We will nurture an innovation project from concept to kitchen table, under one roof. Using our scientific knowhow, application excellence and full portfolio of ingredients, we’ll improve the taste and nutrition profiles of next-generation food and beverages. With our new culinary kitchen, we’re enabling technology focused on the consumer-eating experience, taking food development out of the traditional

PUMA Ambassador and European Champion Gianmarco Tamberi Talks “Only See Great”19.8.2022 11:52:00 CEST | Press release

Six years after his first gold medal, Tamberi takes back the European title and speaks about his motivation to keep performing at the highest level in a video interview with sports company PUMA. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220819005125/en/ Six years after his first gold medal, Tamberi takes back the European title and speaks about his motivation to keep performing at the highest level in a video interview with sports company PUMA. (Photo: Business Wire) With the ‘Only See Great’ campaign, PUMA explores the career path of its brand ambassadors, as they talk about their own pursuit of greatness, listening to their hearts, and finding a vision that no one else can see. In the interview, Gianmarco talks about his very first successes and extraordinary performance at the Olympic Games in Tokyo. “That feeling that I had that day, that happiness I couldn’t even imagine that could exist. It’s something that was co

Kyowa Kirin Receives European Commission Approval for Use of CRYSVITA ® ▼ (burosumab) for the Treatment of Tumour-Induced Osteomalacia (TIO)19.8.2022 10:30:00 CEST | Press release

Kyowa Kirin Co., Ltd. (TSE: 4151, Kyowa Kirin) today announced that the European Commission (EC) approved CRYSVITA® (burosumab) for the treatment of FGF23-related hypophosphataemia in Tumour-Induced Osteomalacia (TIO) associated with phosphaturic mesenchymal tumours (PMTs) that cannot be curatively resected or localised in children and adolescents aged 1 to 17 years and in adults.1 CRYSVITA is also already licensed in the EU for use in the rare disease X-Linked Hypophosphataemia (XLH), for children and adolescents between 1 and 17 years of age with radiographic evidence of bone disease, and in adults.2 Also known as oncogenic osteomalacia, TIO is an acquired disorder caused by typically small, slow-growing, benign PMTs.3,4 It is a rare condition with fewer than 1000 cases reported in the medical literature,4 which mainly affects adults and with a mean onset age of 40 – 45 years.3,5 TIO is associated with progressive and debilitating musculoskeletal deficits,6,7 ultimately having a detr

Kapruvia ® approved in Switzerland with additional regulatory decisions expected in H2 202219.8.2022 07:00:00 CEST | Press release

Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and Cara Therapeutics, Inc. (Nasdaq: CARA) today announced that they have received approval for Kapruvia® from the Swiss Agency for Therapeutic Products (Swissmedic). Kapruvia® will be the first therapy available for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adult hemodialysis patients. Swissmedic approval for Kapruvia® follows approvals by the U.S. Food and Drug Administration, by the European Medicines Agency, by the UK Medicines and Healthcare products Regulatory Agency, as well as by Health Canada. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220818005552/en/ “The approval of Kapruvia® in Switzerland is the next step on our journey to bring this breakthrough treatment to hemodialysis patients living with CKD-associated pruritus around the world,” said Dr. Klaus Henning Jensen, Chief Medical Officer of CSL Vifor. “There is a

European Commission Approves Celltrion Healthcare’s Vegzelma™ (CT-P16, biosimilar bevacizumab) for the Treatment of Multiple Types of Cancer19.8.2022 04:47:00 CEST | Press release

Celltrion Healthcare announced today that the European Commission (EC) has approved Vegzelma™ (CT-P16), a biosimilar bevacizumab referencing EU-approved Avastin®, for the treatment of metastatic breast cancer, non-small cell lung cancer, advanced and/or metastatic renal cell cancer, metastatic carcinoma of the colon or rectum, ovarian cancer and cervical cancer. “The European Commission’s approval of Vegzelma™ will increase access to treatment for patients living with certain types of cancer at an affordable price,” said Kevin Byoung Seo Choi, Senior Vice President and Head of Marketing Division at Celltrion Healthcare. “With proven similarities in efficacy and safety compared to the reference product Avastin®, Vegzelma™ will be available to treat some of the most commonly diagnosed cancers, which collectively affect hundreds of thousands of European patients each year.” The EC approval of Vegzelma™ follows the recommendation for marketing authorisation issued by the Committee for Medi

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom