New drug for children with muscle-wasting disease sets record with $2.125M price tag
On May 24, 2019, the U.S. FDA announced the approval of Zolgensma® (onasemnogene abeparvovec-xioi) as the first gene therapy to treat infants with spinal muscular atrophy (SMA), a rare genetic disease that affects 450-500 infants born in the U.S. annually. When left untreated, SMA can lead to progressive muscle weakness, paralysis requiring permanent ventilation, and death. In clinical trials, Zolgensma® was shown to improve rates of survival, rapid motor function, and milestone achievements, including sitting, walking, and talking. The record-setting price tag of $2.125M is significantly lower than industry predictions but exceeds what the Institute for Clinical and Economic Review (ICER) considers a cost-effective price, according to their analysis. Zolgensma® was approved with Breakthrough Therapy, Fast Track, Orphan Drug, and Priority Review designations.
Researchers warn about serious genital infection in patients on SGLT2 diabetes drugs
In a study published in the Annals of Internal Medicine, a team of researchers identified an increased incidence of Fournier’s gangrene, a rare flesh-eating bacterial infection of the genitals, in patients taking a certain type of diabetic medication, known as SGLT2s. The researchers identified 55 unique cases of Fournier’s gangrene between March 1, 2013, and January 31, 2019 using the FDA’s Adverse event reporting database. To give these numbers historical context, researchers found 19 cases of Fournier’s gangrene between 1984 and January 31, 2019 for patients on other diabetic medications. In the Annals study, researchers noted that it is not possible to establish direct causality between SGLT2s and Fournier’s gangrene but said prescribing doctors should be aware of the possible complication.
From the Industry
New analysis of Phase 3 Mayzent® (siponimod) data shows improvement in think time for MS patients
The new analysis, presented at the American Academy of Neurology (ANN) Annual Meeting, revealed a meaningful improvement in cognitive processing speed (CPS) in patients treated with Mayzent® for Secondary Progressive Multiple Sclerosis (SPMS), an aggressive form of Multiple Sclerosis (MS). CPS is used as a measure of how well a person understands information they receive and changes in CPS are often the first noticeable sign of cognitive function decline in patients with MS. Data from the analysis showed that a significantly higher proportion of patients treated with Mayzent® experienced sustained improvement in CPS versus those on placebo. Impairment in cognitive function is considered one of the most disabling aspects of MS and is estimated to affect 50-70% of patients.
Recent FDA Approvals
New Dosage Form: Nayzilam® (midazolam)
Nasal spray for the treatment of frequent seizure activity in patients with epilepsy 12 years and older [5/17/2019 – Orphan Drug – PROXIMAGEN LLC]
New Active Ingredient: Slynd® (drospirenone)
Oral tablets for use by females of reproductive potential to prevent pregnancy [5/23/19 – EXELTIS USA]
Novel Drug Approval: Piqray® (alpelisib)
Oral tablets to be used in combination with fulvestrant for the treatment of breast cancer [5/24/19 – Priority Review – NOVARTIS PHARMS CORP]
New Generics Entering the Marketplace
Indication: Ulcerative colitis
Dosage Form/Strength: 400MG DR capsules
Average Wholesale Price (AWP): Generic = 400MG ($796) | Brand = 400MG ($838)
Indication: Lung cancer, pancreatic cancer
Dosage Form/Strength: 25MG, 100MG, 150MG tablets
Average Wholesale Price (AWP): Generic = 25MG ($3,101); 100MG ($8,518); 150MG ($9,635) | Brand = 25MG ($3,265); 100MG ($8,966); 150MG ($10,142)