This is the first approved treatment option in over 60 years for patients with the disease.
The US Food and Drug Administration (FDA) has approved Genentech’s rituximab (Rituxan) for the treatment of adults with moderate to severe pemphigus vulgaris (PV), which is a rare and potentially deadly skin disease.
This is the first biologic therapy that has received FDA approval to treat the disease.
“Today’s decision by the FDA provides the first approved treatment option in more than 60 years for patients with pemphigus vulgaris, a potentially life-threatening disease,” Genentech’s chief medical officer and head of Global Product Development Sandra Horning, MD, said in a recent statement. “We are pleased to offer Rituxan as a new and effective therapy to patients with this serious condition.”
Previous to the approval, the drug was granted Priority Review, Breakthrough Therapy Designation, as well as Orphan Designation. Now, with this new approval, the drug is being used to treat 4 different autoimmune diseases.
The approval is based on data collected from a recent phase 3 trial referred to as Ritux. For the randomized, controlled trial, investigators used Roche-manufactured European Union(EU)-approved rituximab in participants. Investigators compared the Ritux 3 regimen (EU-approved rituximab combined with short-term corticosteroids [CS]) with CS by itself as treatment in patients who were newly diagnosed with the disease. Primary endpoint of the study was defined as complete remission at 24 months without the use of steroids for 2 or more months.
Investigators found that 90% of PV patients who were treated with Ritux 3 met the primary endpoint, compared with just 28% of PV patients who were given just CS alone. The findings supported the use of the drug in treating PV patients with moderate to severe disease, while tapering off of CS therapy. The investigators published their results in the March 2017 The Lancet issue.
The International Bullous Disease Consensus Group recently provided new recommendations pertaining to diagnosis and management of the disease. Taking into consideration European treatment guidelines, the panel of experts used a Delphi survey process to achieve international expert consensus. The consensus includes the recommendation to use rituximab along with corticosteroids as first-line therapy for patients with moderate to severe pemphigus.