Four years after a transatlantic slapdown, Merck KGaA will once again seek cladribine OK

Merck Serono CEO Belén Garijo

Merck KGaA is trying to bring cladribine--the multiple sclerosis pill scuttled by regulatory rejections on both sides of the Atlantic four years ago--back from the dead.

According to the company, new data and analysis have stoked their belief that cladribine should be approved, filing notice with the EMA that they'll be resubmitting the drug for approval in Europe. 

Cladribine's failure, spurned by the FDA and the EMA after both regulatory groups concluded that the benefits of this drug did not justify running the safety risks involved in taking it, wound up spurring a massive reorganization of the company. Its Swiss complex was shuttered, thousands were laid off and the company vowed to get back on track, trying to overcome setbacks and poor decisions that have prevented the company from delivering a major new drug product in more than a decade.

In this particular case, the German Merck was once tapped as a leader in the race to develop new oral therapies for MS. But regulators didn't like the data from a single late-stage study, fretting over a possible link to cancer. Now, years later, both Novartis ($NVS) and Biogen ($BIIB) have had years to shore up big markets for rival therapies in the multibillion-dollar MS market.

Over that time, though, regulators have proved much more flexible when it comes to approving new drugs, particularly when clear risk factors are flagged by developers who offer programs to guide careful use. Regulators are also more willing to provide new options for patients who are resistant to existing remedies. PTC Therapeutics ($PTCT) experienced that newfound flexibility first-hand when European regulators did a sudden about-face on their Duchenne muscular dystrophy drug, which has failed in the clinic, and offered a conditional approval.

Merck KGaA hinted about its regulatory strategy in a statement today.

"While the options available to treating neurologists have grown over the years, cladribine tablets have the potential to offer a truly innovative addition to the armamentarium physicians have at their disposal to treat their patients," noted Giancarlo Comi, director of the department of neurology at San Raffaele Hospital in Milan, in a statement for the company.

Merck KGaA has shown a fatal attraction to failed drugs before, though. The company controversially decided to launch a whole new late-stage program for the cancer vaccine Stimuvax, only to dump it after new data proved again that the treatment had only a weak impact. And in recent years, the post of R&D chief at Merck KGaA has seen several directors enter and exit.

In this case, though, the company maintains that the trials that were still ongoing at the time of the rejections and better insights on safety would change the fate of cladribine.

"Time has brought additional data that allow a better characterization of the benefit-risk profile of cladribine, and this has driven our decision to move forward with the registration process," said Belén Garijo, CEO Healthcare.

- here's the release (PDF)

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