Ipsen: The American health authority extends the duration of the examination of an Ipsen molecule


(BFM Bourse) – The Food and Drug Administration has sent a comprehensive response letter regarding the review of palovarotene, a treatment for fibrodysplasia ossificans progressiva. This should not allow Ipsen to receive the opinion of the American health authority before the end of 2023.

Ipsen must take its troubles patiently. The pharmaceutical group is seeking approval in the United States of palovarotene, an experimental drug aimed at treating fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease nicknamed “stone man disease”, which manifests as a extra-skeletal bone formation.

This molecule, resulting from the acquisition of the biotech Clementia in 2019, has experienced clinical development strewn with pitfalls. However, it has already been authorized in Canada and the United Arab Emirates, where it is marketed under the name Sohonos.

For the United States, the approval process is complicated. At the end of October, the Food and Drug Administration (FDA), the American health authority, had decided to postpone indefinitely the meeting of its Advisory Committee on the treatments of endocrine and metabolic diseases concerning the examination of the application for approval of palovarotene , originally scheduled for October 31. The FDA had then requested new information on the clinical trial data, Ipsen specifying that this request did not concern the safety profile of the drug.

Towards a six-month review

On Tuesday, the pharmaceutical company announced that the FDA had this time sent it a “Complete response letter” (CRL) regarding the review of palovarotene.

A CRL is a communication that the FDA sends to pharmaceutical companies to tell them that their dossier cannot be approved as it is.

Ipsen explains that this letter follows the request for additional information that was made in October. “This is not a request for additional data on the efficacy and tolerance of the treatment beyond the scope of existing studies,” assured the company.

However, communication from the FDA will slow down the process. Ipsen said it expects to respond to the FDA’s request in the first quarter of 2023 and anticipates a six-month review period from the US health authority. The group also indicates that the FDA has not yet announced a new date for convening its Advisory Committee on Endocrine and Metabolic Disease Treatments regarding palovarotene.

Better news in 2023?

“While this application extends the review period for palovarotene’s application for approval, we continue to work with the FDA to provide the requested information and remain confident that investigational palovarotene can provide an innovative therapeutic solution to reduce the formation bone outside the normal skeletal system to slow the progression of FOP,” said Howard Mayer, Executive Vice President, Research and Development at Ipsen, quoted in a press release.

On the Paris Stock Exchange, Ipsen lost 0.5% around 10 a.m. while the SBF 120 rose 0.9% at the same time.

UBS included in its forecasts palovarotene sales of around 200 million euros in the United States. The Swiss bank, however, removed them from its model after the FDA made its request for additional information in November.

If Ipsen experiences persistent difficulties with palovarotene and recently suffered a failure in a phase III trial (the last stage of clinical trials before possible commercialization) evaluating cabometyx in the treatment of non-small cell lung cancer, the group could benefit from catalysts over the coming year.

UBS is notably expecting positive results for the phase III trial evaluating elafibranor – which is the subject of a license agreement with the French biotech Genfit – in the treatment of primary biliary cholangitis.

Julien Marion – ©2022 BFM Bourse

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