Regulatory Oncopeptides’ Pepaxto to Remain Sidelined From the U.S. Market

Oncopeptides’ Pepaxto to Remain Sidelined From the U.S. Market


Pepaxto, the multiple myeloma treatment drug from the biotech company Oncopeptides, will remain excluded from the U.S. market, despite being alive and kicking in Europe.

The decision was made in an FDA advisory meeting on Thursday, as oncology experts mulled over whether to reapprove it in the U.S. market. The final vote was 14-2 against the drug, which has been on the market for a year and a half.

Under the FDA’s Accelerated Approval Program – which was formed in 1992 in response to the AIDS crisis – the agency sped up the approval of drugs that treat serious medical conditions. Pepaxto won approval in February of last year under this initiative. The approval was given basd on the results of a 97-patient HORIZON trial.

However, a confirmatory trial showed that it had the potential to be more harmful than beneficial, as a result of which the drug was sidelined from the American market eight months later.

In the OCEAN phase 3 trial – a worldwide open-label study that evaluates the safety of melflufen and dexamethasone in people with multiple myeloma – Pepaxto was found to have a death rate just below 48%, compared to 43.4% for Pomalyst of Bristol Myers Squibb. Another damning statistic was that of the median survival duration; for Pomalyst, it was 25 months, while Pepaxto’s was just 19.7.

Oncopeptides maintains that Pepaxto is quite valuable, at least according to a subgroup analysis which showed positive results when eliminating an important patient group. The company also says that the drug is a better option for elderly people, as opposed to Pomalyst, which takes the cake for younger patients.

Criticizing Oncopeptides for what he termed as a ‘post hoc’ style, Richard Pazdur – founding director of the FDA’s Oncology Center of Excellence – said, “When you have people resubmitting data and changing analysis, this could bring up issues of study conduct and integrity of the study. They have to demonstrate with substantial evidence, efficacy. It is not our responsibility to disprove something.”

After the meeting, Oncopeptides CEO Jakob Lindberg claimed that his company and the FDA couldn’t find a suitable way to collaborate with each other, leading to a complete breakdown in discussions.

The meeting shed further light on why accelerated approval programs have been heavily criticized as of late. The aim of the program was to bring promising life-saving drugs to the market more quickly in the hope of saving more lives. However, it didn’t always work out well, as seen in the case of Biogen’s Alzheimer’s drug Aduhelm, which generated intense controversy as many believed its clinical trial results weren’t conclusive enough to approve it. 

Alexandria Schwarsin, one of the FDA’s clinical reviewers, noted during the meeting that with the information they now have at their disposal, they would not have granted Pepaxto the accelerated approval. That’s because they cannot conclude with absolute certainty that the drug is more beneficial for patients compared to existing therapies.

Lindberg said that while Oncopeptides is not averse to the idea of another confirmatory trial, he understands that funding will be a big issue without sales in the U.S.

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