Novartis scores new FDA nod, breakthrough tag for Promacta

When Novartis paid $16 billion to acquire GlaxoSmithKline’s cancer portfolio back in 2015, it picked up a few drugs that didn’t seem to have quite so clear growth prospects, including Promacta to treat anemia and platelet diseases. But Novartis kept toiling to expand the label for Promacta, which at the time was bringing in about $117 million a year—and there’s new evidence that those efforts are paying off for the company.

Novartis said Friday that the FDA approved Promacta for the first-line treatment of severe aplastic anemia (SAA) in adults and children two years and older, in combination with immunosuppression. It also granted a breakthrough designation to the drug for use in treating acute radiation syndrome, more commonly called radiation sickness.

Sales of Promacta grew 37% year over year in the first nine months of this year, to $844 million. A spokesperson for Novartis didn't provide specifics about how the size of the patient population could grow with the latest label expansion. Severe aplastic anemia is rare: two out of every 1 million people in Europe and North America are diagnosed with the disease each year, he said.

As for radiation sickness, that market is effectively nonexistent, because Promacta would only be used to protect people in the event of a nuclear accident or attack.

"We hope that it will never have to be used," the spokesman admitted. "However, we will be prepared if called upon."

Although Promacta doesn’t address a huge market by any stretch, the SAA label expansion will allow Novartis to reach patients earlier in the treatment process, rather than waiting for them to fail immunosuppressive treatment. The FDA based its approval on an analysis of research performed by Novartis, which showed that 44% of aplastic anemia patients who had not been treated before had a complete response to Promacta combined with immunosuppression at six months. That bested the observed response rate to immunosuppressive drugs alone by 27 percentage points.

"Patients with SAA sometimes do not respond to the current treatment standard," Phillip Scheinberg, M.D., head of the division of hematology at Hospital A Beneficência Portuguesa de São Paulo in Brazil, said in a statement, adding that the ability to add Promacta to immunosuppression upon diagnosis would be valuable to physicians and patients. Scheinberg previously served in the hematology branch of the National Institutes of Health’s National Heart, Lung and Blood Institute (NHLBI), which sponsored Novartis’ research.

Novartis has also filed for approval in Europe for Promacta in first-line SAA. The company said it expects a decision next year.

The news came just a few weeks after Novartis suffered a disappointment in another effort to expand the label of a product that addresses a small patient population. The company had sought approval for its cardiovascular drug Ilaris in the treatment of some patients who had suffered heart attacks. But the agency rejected the label expansion, citing a need for additional clinical trial data on the patients who responded to the drug.

The prospects for a label expansion on Ilaris, which is currently approved to treat rare inflammatory diseases, didn’t seem all that promising last August, when Novartis released clinical trial data showing that the drug produced a 15% reduction in risk for major cardiovascular events and a 10% lower risk of cardiovascular death. Analysts were underwhelmed—and concerned about a risk of infections that seemed higher in the Ilaris group than it was in people taking a placebo.

Disappointments aside, Novartis’ executives remain optimistic about the company’s overall growth prospects. During the company’s third-quarter earnings release in mid-October, they raised their revenue estimate for the year, telling analysts to expect sales to grow by a mid-single digit percentage, rather than the low- to mid-single digit rate they had previously forecast.

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