Novartis' Ilaris nets a trio of rare-disease nods in untapped markets

Novartis HQ cropped

It’s been a while since Novartis’ rare disease med Ilaris picked up a label-expansion boost, but now the Swiss drugmaker has scored three of them.

The pharma giant won FDA go-aheads for the therapy in three distinct types of periodic fever syndromes, a group of autoinflammatory diseases that can prove life-threatening. While all three cover tiny patient populations, they're wide-open markets for the Novartis drug.

Ilaris is now the first and only biologic treatment for tumor necrosis factor-receptor associated periodic syndrome (TRAPS), hyperimmunoglobulin D syndrome (HIDS)/mevalonate kinase deficiency (MKD) and familial mediterranean fever (FMF), Novartis said in a statement.

FMF is the most common of the trio, mainly affecting those of Eastern Mediterranean ancestry. Among those populations, only 1 in 250 to 1 in 1,000 suffer from the malady.

Still,gaining three simultaneous FDA approvals of Ilaris is a momentous turning point for people who suffer with these severe, debilitating diseases," Novartis Pharmaceuticals CEO Paul Hudson said in a statement, citing “significant unmet need ... particularly in children.”

Previous treatments for the trifecta of rare conditions consisted of oral anti-inflammatory drugs--such as corticosteroids--or nonsteroidal anti-inflammatory meds, but those only helped manage symptoms, rather than preventing flare-ups in the first place.

Ilaris, on the other hand, showed in a Phase III study that through 16 weeks, it could top placebo at controlling disease in patients with any of the three diseases. With those data, Ilaris nabbed med three priority review tags from the FDA, which sped the new indications through the approval process.

Before the spate of approvals, Ilaris had indications for cryopyrin-associated periodic syndromes (CAPS)--another periodic fever syndrome condition--and systemic juvenile idiopathic arthritis (SJIA) on its resume, with thumbs-ups coming in mid-2009 and mid-2013, respectively.

And while the drug has hardly been a top seller for the Basel-based drugmaker, it’s been growing as of late: In 2015, it put up $236 million in sales, a 19% increase over 2014’s haul.

Special Report: Top 20 orphan drugs by 2018

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