Mallinckrodt plc , a leading global specialty pharmaceutical company, announced it will conduct a new phase 4, multi-center, randomized, double blind, placebo-controlled study to further assess the efficacy of H.P. Acthar gel as a therapy option in patients with symptomatic sarcoidosis, one of 19 indications approved by the US Food and Drug Administration (FDA).

"In my experience, a considerable number of sarcoidosis patients can have persistent disease that may not be resolved by first line treatment," said Dr. Robert Baughman, M.D., Professor from Department of Internal Medicine, University of Cincinnati Medical Center, Cincinnati, Ohio. "The results from this sarcoidosis phase 4 study in this patient population can potentially provide substantial evidence to further support Acthar as a treatment option in appropriate symptomatic sarcoidosis patients."

"We are pleased to announce our plans to initiate this study of the efficacy of Acthar therapy as a later line treatment option for sarcoidosis patients with persistent disease," said Steven Romano, M.D., chief scientific officer and executive vice president at Mallinckrodt. "H.P Acthar gel is new to many pulmonologists and the new phase 4 study – a 'pilot' study in 100 patients – is being designed as an exploratory evaluation of a subset of the more challenging patients, many of whom may not have responded to multiple previous therapies. The study will help us better understand and outline for physicians which patients may benefit from a treatment alternative like Acthar – perhaps those who aren't responding to traditional treatments."

Sarcoidosis is a challenging, yet treatable, rare multisystem disease. In some cases the symptoms may come and go throughout a lifetime. This is referred to as symptomatic sarcoidosis. In people with sarcoidosis the immune system overreacts, forming clumps of cells called granulomas that result in inflammation to the body's tissues. The disease can impact any organ, but it most often impacts the lungs, lymph nodes, eyes, and skin. Over 90 per cent of people with sarcoidosis will suffer lung problems. Concomitant involvement of organs outside of the lungs is common, occurring in as many as half of all sarcoidosis cases.

HP Acthar gel is an injectable drug approved by the FDA for the treatment of 19 indications. Of these, today the majority of Acthar use is in these indications: Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus; Monotherapy for the treatment of infantile spasms in infants and children under 2 years of age;     The treatment of acute exacerbations of multiple sclerosis in adults. Controlled clinical trials have shown H.P. Acthar Gel to be effective in speeding the resolution of acute exacerbations of multiple sclerosis. However, there is no evidence that it affects the ultimate outcome or natural history of the disease; Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus; Treatment during an exacerbation or as maintenance therapy in selected cases of systemic dermatomyositis (polymyositis); The treatment of symptomatic sarcoidosis;  Adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy);  Treatment of severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation.