Rare Disease Day 2025 triggered a flurry of pharma marketing activity, with Arrowhead Pharmaceuticals, Zevra Therapeutics and a clutch of other drug developers publishing materials to mark the Feb. 28 event.
Zevra used the awareness day to roll out the “Learn NPC, Read Between the Signs” disease awareness campaign. The biotech won FDA approval for a Niemann-Pick disease type C (NPC) treatment, Miplyffa, last year and recently agreed to sell a priority review voucher for $150 million to fund the launch and other work.
The new campaign is intended to support the early identification of NPC, an ultra-rare disease that can be hard to diagnose. People develop NPC at various ages and can present with different symptoms, and that heterogeneity can delay diagnosis. Zevra’s LearnNPC.com website features resources intended to enable early detection of NPC, including a table that shows the symptoms that are typical at different ages.
Arrowhead, meanwhile, marked Rare Disease Day by launching a new patient resource hub. The hub, which builds on the biotech’s “We’ll Get There Soon” campaign, is designed to provide familial chylomicronemia syndrome (FCS) patients with education, tools and support.
Visitors to LowerMyTGs.com can learn about the association between triglyceride levels, which are high in people with FCS, and the risk of pancreatitis flares. Arrowhead has developed the siRNA plozasiran to bring triglyceride levels down below the 500 mg/dL risk threshold. And, with the FDA set to rule on a filing for approval in November, the biotech has begun to lay the groundwork for a planned launch.
Several other biopharma companies also commemorated Rare Disease Day, which is celebrated on the last day of February every year.
Avidity highlighted its sponsorship of a Duchenne muscular dystrophy webinar, while Abionyx noted its support for the creation of a European association of patients with LCAT deficiency. Sobi focused on the need for global partnerships and long-term commitment, and Servier detailed its rare disease R&D programs.
AstraZeneca’s rare disease unit, Alexion, and Harmony Biosciences joined an episode of the morning show “The Balancing Act” to highlight the experiences of those living with rare diseases and the importance of boosting diagnoses and awareness. CSL once again participated in the popular “Light Up for Rare” movement that calls for homes and landmarks to be adorned in lights on the night of Rare Disease Day.
And, in a blog post, Ipsen’s chief corporate affairs officer, Josep Catlla, wrote about the company's work to create “a future where barriers are broken down, and the potential for life-changing innovation is fully realised” for the rare disease community.