Clinigen gets orphan drug designation for 'aldesleukin' in the US

Pharmaceutical services company Clinigen Group announced on Monday that the US Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted orphan drug designation for ‘aldesleukin’, in the treatment of amyotrophic lateral sclerosis (ALS).

The AIM-traded firm described ALS as a severe neurodegenerative disease which affects motor neurons leading to progressive muscle weakness, paralysis and ultimately death within a median time of two to four years from disease onset.

It said orphan drug designation in the US recognised the potential therapeutic role of aldesleukin in the disease, and could provide a number of benefits for Clinigen should it obtain a marketing approval for the indication.

Those benefits would include seven years’ marketing exclusivity within the US on launch, along with tax credits for clinical development costs and fee waivers.

Studies in ALS patients had shown that decreased levels of regulatory T-cells (Tregs) were associated with increased disease severity, and predictive of disease progression and survival, suggesting that Tregs could be an attractive therapeutic target.

Aldesleukin, when administered at doses significantly lower than those used in oncologic indications, had been shown to enhance Treg function in inflammatory and auto-immune conditions, Clinigen explained.

It said it was supplying aldesleukin being used in the ongoing ‘MIROCALS’ study, evaluating its clinical potential within ALS and is investigating the optimal pathway to generating the data required to support an application for a marketing authorisation.

“The orphan drug designation issued by the FDA recognizes the potential of aldesleukin as a possible valuable new treatment for patients with ALS where there is a significant level of unmet need within the disease area,” said group chief executive officer Shaun Chilton.

“Clinigen is also exploring the use of aldesleukin in several other therapeutic areas where its modulatory effects on Tregs may have a beneficial clinical effect.”

At 1627 BST, shares in Clinigen Group were up 2.05% at 796.5p.