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Monthly News Roundup - May 2019

Zolgensma: First Gene Therapy for Pediatric Spinal Muscular Atrophy (SMA)

Zolgensma (onasemnogene abeparvovec-xioi) from AveXis (a Novartis company), is now approved for children less than 2 years of age with spinal muscular atrophy (SMA). SMA is a rare genetic motor neuron disease and most untreated children do not survive past the age of two due to respiratory failure.

  • Zolgensma is given as a single, one-time infusion and replaces the defective or missing survival motor neuron gene (SMN1) to treat the root cause of SMA and halt disease progression.
  • In an ongoing clinical trial, effectiveness was demonstrated in 36 patients who were between 2 weeks and 8 months old at study entry. Of 21 patients treated with Zolgensma, there are 19 remaining patients, who range in age from 9.4 to 18.5 months; 13 of these 19 patients are at least 14 months of age. Developmental motor milestones were also met.
  • Zolgensma can cause acute serious liver injury. In studies, the most common side effects were elevated liver enzymes and vomiting.
  • Total cost is $2.125 million, according to AveXis, and is considered to be the world's most expensive single-dose, one-time drug. For reimbursement assistance, call 1-855-441-GENE (1-855-441-4363).

Novartis’ Piqray Approved as First PI3K Inhibitor for Breast Cancer

The FDA has approved Novartis’ Piqray (alpelisib) tablets, a kinase inhibitor to be used in combination with the endocrine therapy fulvestrant (Faslodex) to treat postmenopausal women, and men, with HR-positive, HER2-negative, PIK3CA-mutated, advanced or metastatic breast cancer.

  • In the Phase 3 SOLAR-1 study, 341 patients were enrolled with a PIK3CA mutation. Results showed that Piqray + fulvestrant, when compared to placebo + fulvestrant, significantly prolonged progression-free survival (PFS, median of 11 months vs. 5.7 months, respectively) in patients whose tumors had a PIK3CA mutation.
  • No PFS benefit was observed in patients whose tumors did not have a PIK3CA tissue mutation.
  • Common side effects include high blood sugar levels, elevated creatinine, diarrhea, rash, and hair loss, among others. Severe cutaneous reactions may also occur.

FDA Clears Nayzilam as First Nasal Spray to Treat Seizure Clusters

This month the FDA approved UCB's Nayzilam (midazolam) nasal spray, a single-dose benzodiazepine used for acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients 12 years of age and older.

  • Nayzilam can be given by a non-healthcare provider such as a caregiver or family member.
  • In a Phase 3 study, 201 patients were on stable seizure treatment but experiencing seizure clusters. A significantly greater percent of patients met the primary endpoint, defined as termination of seizure(s) within 10 minutes after the initial dose of Nayzilam 5 mg or placebo (80.6 versus 70.1%) and the absence of seizure recurrence between 10 minutes and 6 hours after the initial dose (58.2 versus 37.3%).
  • The most common side effects were drowsiness (somnolence), headache, nasal discomfort, throat irritation, and runny nose (rhinorrhea).

Ruzurgi Approved for Pediatric Lambert-Eaton Myasthenic Syndrome (LEMS)

The FDA has approved Ruzurgi (amifampridine) tablets, the first approval specifically for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to under 17 years of age. Ruzurgi is a potassium-channel blocker.

  • LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms often affecting activities of daily living.
  • Pediatric use of the drug is supported by evidence from a placebo-controlled withdrawal study in 32 adults with LEMS that had taken the drug for at least 3 months. Results demonstrated less impairment in a test of walking.
  • Common side effects were similar to adults and included burning or prickling sensation (paresthesia), stomach pain, indigestion, dizziness and nausea.

FDA OKs Vyndaqel and Vyndamax as First Treatments for Rare Heart Disease

In May, the FDA approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of cardiomyopathy (a form of heart disease) of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults.

  • These are the first FDA-approved treatments for ATTR-CM.
  • Vyndaqel and Vyndamax have the same active ingredient, tafamidis, but cannot be substituted on a milligram to milligram basis because their recommended doses differ.
  • In studies after a period of 30 months, the survival rate was higher in the Vyndaqel group than in the placebo group and hospitalization rates were lower. Tafamidis may cause fetal harm in pregnancy.
  • Approvals were granted to FoldRx, a subsidiary of Pfizer.

Posted: May 2019