Actemra a breakthrough for systemic sclerosis, says FDA

Roche's rheumatoid arthritis (RA) drug Actemra/RoActemra could be an important therapy for the life-threatening autoimmune disease systemic sclerosis (SSc), according to the US FDA.

The US regulator has just awarded breakthrough status to Actemra (tocilizumab) for SSc, also known as systemic scleroderma, which is characterised by skin thickening and, in severe cases, damage to the circulatory system and organs.

SSc has the highest mortality rate of any rheumatic disease, according to Roche, which notes that there are no approved disease-modifying drugs for the condition. Patients are typically managed with drugs to treat symptoms, including emollients for itchy and flaky skin, ACE inhibitors for those with kidney complications and calcium antagonists in cases where the disease affects the lungs.

Roche has started preparing for a placebo-controlled phase III trial of Actemra in patients with SSc. Recruitment is due to get underway in September, with an enrolment target of 210 subjects, and the trial is due to complete in 2019.

The news comes just as the European League against Rheumatism (EULAR) annual conference gets underway in Rome, at which Roche will present new data from a phase II study of Actemra in SSc called faSScinate.

The results – due for presentation tomorrow – will show that, while the primary outcome objective in the trial was not reached, there was a positive trend towards efficacy in terms of skin thickening that warrants further study, according to Roche. The 48-week trial is using the Rodnan skin score as a primary endpoint.

Actemra was first launched a decade ago for rheumatoid arthritis (RA) and has been a big success for the Swiss pharma major. An extension of its approved indications to include early RA in the EU last year and approval of a subcutaneous formulation to replace the older infusion helped the drug grow 23 per cent to 1.22 billion Swiss francs, making it one of the fastest growing drugs in Roche's portfolio.

Roche is also developing its Esbriet (pirfenidone) product as a treatment for interstitial lung disease in patients with SSc, with a phase II trial ongoing. Esbriet is already approved to treat the deadly lung disease idiopathic pulmonary fibrosis (IPF).

The incidence of SSc is difficult to measure, but it is estimated to affect approximately 2.5 million people worldwide, the company states.

Meanwhile, Roche will also present new data at EULAR 2015 on Actemra in early-stage RA showing almost double sustained remission rates in recently-diagnosed patients, as well as results showing sustained efficacy in systemic juvenile idiopathic arthritis after five years' treatment with the drug.