Many patients with rare conditions know that sometimes it can be extremely difficult to find targeted treatment options. In fact, a large number of rare conditions still have no approved therapies. However, that might be changing for patients with neuromyelitis optica spectrum disorder (NMOSD), according to Healio. Between June 2019 and October 2020, the FDA approved three new therapies for patients with NMOSD.
The first of the three FDA-approved therapies is Soliris (eculizumab). Developed by Alexion Pharmaceuticals (“Alexion”), Soliris is indicated for patients with anti-AQP4 antibody-positive NMOSD, which occurs in around 75% of patients. Patients with this form of NMOSD experience an overactive complement system. Since the complement system plays a role in inflammation, an overactive complement system can cause nerve, brain, and spinal cord damage. Soliris works by inhibiting a complement protein called C5, reducing this activation and preventing NMOSD relapse.
Data from the Phase 3 PREVENT clinical trial showed that 98% of patients taking Soliris experienced no relapses for 48 weeks (11 months) or more. Patients first receive 900mg Soliris for 4 weeks, followed by 1,200mg bi-weekly. Because of the potential for meningococcal infections, patients taking Soliris should be vaccinated prior to treatment.
In June 2020, the FDA approved Uplizna (inebilizumab-cdon injection). Developed by Viela Bio (“Viela”), Uplizna also received both Orphan Drug and Breakthrough Therapy designations. Its approval hinged on the N-MOmentum clinical trial, which enrolled 230 patients. During the trial, Uplizna reduced the risk of relapse in patients with anti-AQP4 antibody positive NMOSD by 77%. Additionally, 89% of patients had no relapse for 6 months, compared to only 58% of those who received a placebo. Generally, Uplizna was safe, effective, and well-tolerated. However, side effects included a cold, headache, and urinary tract infections (UTIs).
Finally, Enspryng (satralizumab-mwge) became FDA-approved in August 2020. Developed by Genentech, Enspryng is the only NMOSD treatment that is subcutaneously administered. It targets and inhibits interleukin-6 (IL-6), a pro-inflammatory cytokine. Patients receive an initial loading dose, followed by an injection every 4 weeks. Over the first year of treatment, patients require around 15 doses. In the Phase 3 SAkuraStar and SAkuraSky trials, Enspryng reduced relapses in patients.
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Also known as Devic’s syndrome or Devic disease, neuromyelitis optica is an autoimmune disorder in which the immune system mistakenly attacks nerve tissue on the spine and optic nerve. In some cases, it is confused with or misdiagnosed as multiple sclerosis. An estimated 4,000-15,000 American patients have NMOSD. The condition usually occurs in attacks, punctuated by periods of remission. Symptoms include:
- Appetite loss
- Muscle spasms
- Blurred vision
- Inability to recognize colors
- Complete vision loss
- Loss of bowel and bladder control
- Limb weakness
- Burning or tingling sensations in the legs
Learn more about NMOSD.