Indication Expansion for Soliris
On Feb. 28, 2025, the US Food and Drug Administration (FDA) approved Soliris® (eculizumab – Alexion/AstraZeneca) for pediatric patients six years of age and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. Caused by an autoimmune reaction that damages parts of muscles where nerve signals are received, gMG results in muscle weakness. The eyes and face are particularly affected for most patients, and around one patient in 10 may have potentially fatal involvement of muscles used to breathe. A monoclonal antibody that is a complement inhibitor, Soliris has been approved for the treatment of adults with gMG since 2017, but this pediatric expansion marks the first treatment available for pediatric patients living with gMG. Clinical trials showed comparable efficacy and safety data to adults. Dosing is weight based for this population and is administered via intravenous infusion (IV) over one to four hours for pediatric patients while only over 35 minutes for adults. Soliris also has indications for other rare diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and neuromyelitis optica spectrum disorder (NMOSD). It is available only through a risk evaluation and mitigation strategy (REMS). Its labeling contains a boxed warning about an increased risk of serious meningococcal infections associated with its use. Patents should be vaccinated against meningitis at least two weeks before beginning treatment with Soliris and they should be monitored for signs of meningococcal infections during therapy.
New Indication for Iluvien
Ani Pharmaceuticals announced the FDA approval of Iluvien® (fluocinolone acetonide intravitreal implant) for the treatment of chronic non-infectious uveitis affecting the posterior segment of the eye (NIU-PS) on March 14, 2025. Initially approved in 2014 for the treatment of diabetic macular edema (DME), this implant is injected into the back of the eye and releases fluocinolone acetonide, a corticosteroid, for 36 months. NIU-PS is an inflammatory disease of the eye and can lead to permanent vision loss. There were also labeling updates made in the Warning and Precautions section about corticosteroid use, including delayed healing after cataract surgery and perforation in diseases causing thinning of the cornea or sclera.
Mercaptopurine Oral Suspension Approved and Launched
On Feb. 27, 2025, the FDA approved Hikma Pharmaceutical’s mercaptopurine oral suspension in a 20mg/mL dose for the treatment of acute lymphoblastic leukemia (ALL). Although generic mercaptopurine 50mg tablets are available, this is the first generic to Rare Disease Therapeutics’ Purixan® (mercaptopurine) oral suspension formulation and therefore has 180 days of Competitive Generic Therapy exclusivity. According to IQVIA, US sales of mercaptopurine oral suspension were $14 million in 2024. Pricing will vary since the dose is weight based; however, it may range from $1,633 to $4,900 per month, about 5% lower in cost than the brand. The generic was launched on Feb. 27, 2025.