Novartis wins an FDA nod for a long-acting spin on its rare disease drug

The FDA signed off on a once-a-month version of Novartis' ($NVS) Signifor designed to treat acromegaly, a rare growth hormone disorder that can lead to disfigurement and death.

The treatment, injected intramuscularly, binds to the body's somatostatin receptors to tamp down the excess growth hormone and insulin-like growth factor-1 (IGF-1) production at the root of the disease, reducing acromegaly's trademark swelling of the hands and face and improving patient outcomes, Novartis said. In two Phase III trials, the orphan drug met its goal of regulating growth hormone and IGF-1 levels in patients either treatment naïve or taking last-genearation somatostatin analogs.

With the approval, Signifor, already approved to treat Cushing's disease in a twice-daily formulation, will take the mantle from Novartis' own Sandostatin, a blockbuster injectable for acromegaly that came off patent in the U.S. this year. The disease is estimated to effect about 60 of every million people worldwide, Novartis said, but, due to difficulties in diagnosis, that number could be nearly 5 times greater.

"The FDA approval of Signifor LAR for acromegaly marks an important day for physicians and patients living with difficult-to-treat pituitary conditions and underscores our continued commitment to helping patients manage rare diseases," Novartis Oncology President Bruno Strigini. "We are pleased that a new treatment option is now available to help address the serious impact of uncontrolled acromegaly, and are optimistic about providing this much needed treatment to other patients worldwide in the near future."

But Novartis isn't the only contender in the acromegaly field. Roche ($RHHBY) reached out to Chiasma in a $595 million deal last year to get its hands on a Phase III oral treatment for the disease, which the companies believe could disrupt the injectable-dominated market.

Separately, Novartis inked a deal with Israel's BioLineRx ($BLRX), trading $10 million for a 12.8% stake in the company and the right to collaborate on early-stage projects. Under the agreement, BioLineRx will pitch candidates to Novartis which can buy in for $5 million per drug, agreeing to fund 50% of development costs with a right to license.

Meanwhile, the Swiss drugmaker is pouring money into its promising pipeline, last year spending nearly $9.9 billion, 17% of its total revenue, on R&D. And Novartis has big expectations for that pipeline, expecting to hit the $1 billion annual sales mark for 14 of its candidates in the next four years.

- read the Signifor news
- get more on the BioLineRx deal

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