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Monthly News Roundup - March 2021

FDA OKs Janssen’s Ponvory for Relapsing Forms of Multiple Sclerosis

The U.S. Food and Drug Administration (FDA has approved Janssen’s once-daily oral Ponvory (ponesimod) tablets to treat relapsing forms of multiple sclerosis in adults, including clinically isolated syndrome, relapsing remitting disease, and active secondary progressive disease.

  • Ponvory is classified as a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator and is thought to bind to key receptors on white blood cells (lymphocytes) in the immune system, slowing the spread of brain and spinal cord nerve damage.
  • In a Phase 3 head-to-head clinical trial with teriflunomide (Aubagio) in 1,133 participants, Ponvory significantly reduced MS annualized relapses (Ponvory 71% vs. Aubagio 61%), and was superior to Aubagio in reducing the number lesions. Differences in rates of disability progression were not statistically significant between the Ponvory and Aubagio groups.
  • The recommended maintenance dosage is 20 mg once daily. Ponvory does not require genetic testing or first-dose cardiac monitoring for most patients.
  • Common side effects (occurring in at least 10% of patients) include upper respiratory tract infection, liver enzyme elevation, and high blood pressure. 

Bristol Myers Squibb’s Abecma Approved as First CAR-T Cell Therapy for Multiple Myeloma

In March, the FDA approved Abecma (idecabtagene vicleucel), the first B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell immunotherapy.

  • Abecma is indicated for the treatment of adults with multiple myeloma who have not responded to, or whose disease has returned after, four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.
  • CAR-T cell therapy is made from a patient’s white blood cells that are genetically modified. Abecma is approved as a one-time infusion given in a certified healthcare facility. Abecma recognizes and binds to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.
  •  In a Phase 2 study of 100 evaluable patients, 72% of patients partially or completely responded, with 28% showing a complete response (disappearance of all signs of multiple myeloma).
  •  Common, serious and fatal side effects can occur with Abecma. Cytokine release syndrome (CRS), neurologic toxicity, and other serious and possibly fatal toxicities are outlined in the Boxed Warning.
  • Other approved CAR-T cell therapies include: Breyanzi, Kymriah, Tecartus and Yescarta.

Zegalogue OK’d for Severe Low Blood Sugar in People with Diabetes 

Severe hypoglycemia is an acute, life-threatening condition resulting from a critical drop in blood glucose (sugar) levels linked primarily with insulin therapy. In response, the FDA has now approved Zegalogue (dasiglucagon) injection for the treatment of severe hypoglycemia (low blood sugar) in patients with diabetes aged 6 years and above.

  • Zegalogue works by increasing blood glucose concentration through activation of hepatic glucagon receptors, thereby stimulating glycogen breakdown, and release of glucose from the liver.
  • Approval was based on Phase 3 studies in children aged 6 to 17 years and in adults with type 1 diabetes. The primary endpoint, time to plasma glucose recovery (treatment success) was defined as an increase in blood glucose of ≥20 mg/dL from time of administration, without additional intervention within 45 minutes. The results were significant with Zegalogue (10 minutes) vs. placebo (30-45 minutes) across both age populations.
  • Common side effects (≥ 2%) include nausea, vomiting, headache, and injection site pain. Diarrhea was also reported in adults.
  • Zegalogue from Zealand Pharma is available as a single-dose autoinjector and prefilled syringe and is expected to be commercially available in June.

Fotivda Approved to Treat Advanced Kidney (Renal Cell) Cancer

This past month the FDA approved oral Fotivda (tivozanib) for the treatment of adults with relapsed or refractory advanced renal cell carcinoma (RCC) who have received two or more prior systemic therapies.

  • Fotivda, from AVEO Oncology, is classified as a vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI).
  • In the Phase 3 TIVO-3 study, 350 participants were randomized to receive either Fotivda or sorafenib. The main efficacy outcome, progression-free survival (PFS), was 5.6 months (95% CI: 4.8, 7.3) in the Fotivda arm compared with 3.9 months (95% CI: 3.7, 5.6) for those treated with sorafenib (HR 0.73; 95% CI: 0.56, 0.95; p=0.016).
  • Fotivda is taken once daily with or without food for 21 days every 28 days on-treatment, followed by 7 days off-treatment (28-day cycle) until disease progression or unacceptable toxicity.
  • Common side effects are fatigue, high blood pressure (hypertension), diarrhea, decreased appetite, nausea, dysphonia (difficulty speaking), hypothyroidism (low thyroid levels), and cough, among others.

FDA Approves Azstarys for the Once-Daily Treatment of ADHD

Attention Deficit Hyperactivity Disorder (ADHD) is a behavioral disorder that can result in the inability to focus, hyperactivity, and impulsivity and hasty actions without thought. An estimated 8.4% of children and 2.5% of adults have ADHD.

  • The FDA has now approved KemPharm’s Azstarys (dexmethylphenidate and serdexmethylphenidate), a central nervous system (CNS) stimulant for the treatment of ADHD in patients 6 years of age and older.
  • Azstarys contains serdexmethylphenidate, a prodrug of dexmethylphenidate, co-formulated with immediate-release dexmethylphenidate. This combination provides an early onset and a longer duration of action without the spikes in dexmethylphenidate concentrations. Azstarys capsules are taken once daily with or without food.
  • Common side effects include decreased appetite, insomnia, nausea, vomiting, dyspepsia (heartburn), stomach pain, and decreased weight, among others.

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