Priovant Therapeutics has secured approval from the US Food and Drug Administration (FDA) for its oral therapy Lisraya (brepocitinib) in a rare autoimmune disease called dermatomyositis, becoming the first targeted therapy authorised for this condition.
Based on the FDA’s verdict, the once-daily, dual tyrosine kinase 2 (TYK) and Janus kinase 1 (JAK1) inhibitor will now become available to American adults with dermatomyositis regardless of their disease activity levels, prior treatment experience or clinical presentation. Priovant claims it will make the drug available to patients immediately.
The FDA has also left it up to prescribers to decide how Lisraya is used, as the agency has deemed it suitable as both an add-on therapy and alternative therapy to non-targeted treatments such as steroids, unspecific immunomodulators and intravenous immunoglobulin (IVIg).
Lisraya’s US approval stems from the positive outcomes of the Phase III VALOR trial (NCT05437263), in which the drug offered significant improvements across multiple dermatomyositis disease domains as early as week four.
Dermatomyositis is a rare, systemic autoimmune condition where a patient’s antibodies target both the skin and muscles – resulting in progressive weakness and itchy and painful skin lesions. Currently, researchers estimate that around 18,850 people in the US are currently living with the condition.
Alongside its efficacy in reducing symptoms of the condition, Lisraya also diminished steroid dependency amongst treated patients, with 45% coming off steroids completely following a 52-week course of the treatment compared with 29% in the placebo group. Healthcare professionals generally prescribe steroids as part of standard treatment for dermatomyositis, though their long-term use carries a high risk of side effects.
According to Ruth Ann Vleugels, founding director of the Autoimmune Skin Disease Center and Connective Tissue Disease Clinics at Mass General Brigham, this approval marks a “turning point” for patients with dermatomyositis that can offer “meaningful benefit across muscle, skin and overall disease activity while simultaneously reducing reliance on systemic corticosteroids.”
Dermatomyositis is not the only indication Priovant has its eye on for Lisraya, however, as the drug is currently in late-stage development for a non-infectious eye condition uveitis, as well as skin and scalp conditions, cutaneous sarcoidosis, and lichen planopilaris.
Facilitating access to Lisraya
With Lisraya’s approval in the bag, Priovant is now looking to ensure that patients can start treatment as quickly as possible, as accessibility challenges continue to plague rare disease communities on a global scale.
To achieve this, Priovant has established a distribution network of specialty pharmacies, while creating a personalised assistance platform to help patients with insurance coverage, treatment support and securing financial assistance. According to Priovant, through this system, patients may pay as little as $0 per month for treatment with Lisraya.
According to a recent report from the National Organization for Rare Disorders (NORD), access to rare disease therapies in the US remains variable on the state level, with patients in certain regions of the country not receiving adequate protection from short-term “junk” insurance plans that can exclude key treatments and benefits.
