Novartis scores recommendation from EMA for myeloma drug Farydak

Novartis ($NVS) has had a tough road with its multiple myeloma med Farydak (panobinostat) in the U.S., with the FDA last year waving off a speedy review process for the drug in light of safety concerns. But Farydak has recovered some since then, scoring FDA approval earlier this year after the agency decided to do an about-face on the med. Now the drug is picking up steam across the pond, snagging the European Medicines Agency's (EMA) recommendation for patients with advanced forms of the disease.

The EMA's Committee for Medicinal Products for Human Use (CHMP) signed off on Farydak for patients with relapsed or refractory forms of the disease who have already undergone at least two treatments, including Takeda's blockbuster Velcade and an immunomodulatory agent, the agency said in a statement. The drug should be used in combination with Velcade and the steroid dexamethasone, it added.

The CHMP based its recommendation on a study that looked at Farydak in combination with Velcade and the steroid dexamethasone in 768 patients with multiple myeloma. Of the 768 patients included in the study, 193 had already been treated for the disease with Velcade and an immunomodulatory agent. Individuals who received Farydak in addition to the other drugs had a progression-free survival rate of 10.6 months, compared to 5.8 months for patients who only took Velcade and dexamethasone.

But Farydak also comes with some serious side effects, including blood disorders, hemorrhage, diarrhea and vomiting, the EMA pointed out, so the agency is requiring a follow-up plan to monitor the drug's safety and lower its risks.

The positive recommendation echoes the FDA's decision on Farydak earlier this year. In March, the agency shrugged off the advice of its panel of independent experts and decided to approve the drug for use with Velcade and dexamethasone in patients who have failed treatment twice. But regulators also saddled Farydak with a black-box warning, cautioning against risks of severe and often fatal cardiac side effects.

Now, with good news for its drug in the U.S. and Europe, Novartis is better positioned to battle it out with rivals in the multiple myeloma market, including Amgen's ($AMGN) Kyprolis and Celgene's ($CELG) Pomalyst. Still, Novartis will have to work hard for its share of the pie as Kyprolis and Pomalyst trot out some promising numbers. Amgen earlier this year announced interim study results which showed that patients taking Kyprolis and dexamethasone lived for a median of 18.7 months without their disease worsening, almost double the 9.4 months that Velcade posted. And Kyprolis doesn't have Farydak's safety issues.

Both Kyprolis and Farydak will have some serious ground to cover to catch up with Pomalyst, which brought in $679.7 million in sales in 2014. Kyprolis posted less than half that amount with $306 million in sales last year.

- read the EMA's statement

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