FDA gives fast-track approval to new ALS drug
The FDA has approved the first treatment that takes a genetics-based approach to slowing or stopping the progression of a rare form of amyotrophic lateral sclerosis (ALS).
The FDA fast-tracked the approval of Qalsody (Biogen) based on early trial results. The agency said in a news release that its decision was based on the demonstrated ability of the drug to reduce a protein in the blood that is a sign of degeneration of brain and nerve cells.
Qalsody is given to people via a spinal injection, with an initial course of three injections every 2 weeks. People then get the injection once every 28 days.
The new treatment is approved only for people with SOD1-ALS, which is known for a genetic mutation. While ALS affects up to 32,000 people in the United States, just 2% of people with ALS have the SOD1 gene mutation. The FDA says the number of people in the United States who could use Qalsody is about 500.
FDA OKs first treatment for Friedreich ataxia
The FDA has approved the first treatment for the neurodegenerative disorder Friedreich ataxia for use in adults and adolescents aged 16 and older.
The recommended dose of omaveloxolone (Skyclarys, Reata Pharmaceuticals) is 150 mg (three capsules) taken orally once daily on an empty stomach.
The FDA approval of omaveloxolone was supported by a randomized double-blind, placebo-controlled study comprising 103 patients with genetically confirmed Friedreich ataxia and baseline modified Friedreich Ataxia Rating Scale (mFARS) scores between 20 and 80.
Treatment with the novel medication led to statistically significant lower mFARS scores, signifying less impairment, relative to placebo, at week 48. The placebo-corrected difference between the two groups was –2.41 points (P = .0138).
Omaveloxolone received priority review and had orphan drug, fast track, and rare pediatric disease designations.
FDA OKs first drug for Rett syndrome
The FDA has approved trofinetide oral solution (Daybue, Acadia Pharmaceuticals) as the first treatment of Rett syndrome in adults and children aged 2 years and older.
Trofinetide is a synthetic analogue of the amino-terminal tripeptide of insulinlike growth factor-1, which occurs naturally in the brain. The drug is designed to treat the core symptoms of Rett syndrome by potentially reducing neuroinflammation and supporting synaptic function.
The approval of trofinetide was supported by results from the pivotal phase 3 LAVENDER study that tested the efficacy and safety of trofinetide versus placebo in 187 female patients with Rett syndrome, aged 5-20 years.
More data back Guillain-Barré risk with Janssen COVID shot
New surveillance data from the Vaccine Adverse Event Reporting System (VAERS) back previous findings of increased risk for Guillain-Barré syndrome (GBS) after receiving the Janssen COVID-19 vaccine (Ad26.COV2.S).
Over 14 months, GBS reporting rates within 21 and 42 days of administration of Janssen’s replication-incompetent adenoviral vector vaccine were approximately 9-12 times higher than after administration of the Pfizer-BioNTech (BNT162b2) or the Moderna (mRNA-1273) mRNA COVID vaccines.
Additionally, observed GBS cases after the Janssen shot were two to three times greater than expected, based on background rates within 21 and 42 days of vaccination.
Conversely, and confirming prior data, there was no increased risk for GBS with the Pfizer or Moderna vaccines and no significant difference between observed and expected numbers of GBS cases after either mRNA COVID-19 vaccine.
The findings were published online in JAMA Network Open (2023 Feb 1. doi: 10.1001/jamanetworkopen.2022.53845).