Tagged News
FDA's New Real-World Data Guidance: Streamlining Drug Development and Regulatory Approval
- The FDA has released new guidance clarifying that non-interventional studies using real-world data (RWD) do not require investigational new drug applications, potentially accelerating drug development timelines.
- The guidelines address three key challenges: long-term patient follow-up after clinical trials, participants lost to follow-up during trials, and demonstrating value for reimbursement of novel therapies.
- By incorporating RWD from sources like electronic health records, claims data, and registries, pharmaceutical companies can enhance clinical trial designs and provide more comprehensive evidence for regulatory decision-making.
First Veteran Receives FDA-Approved Qalsody for SOD1-ALS at Houston VA Medical Center
- Roy Swearingen, a 65-year-old Marine Corps veteran from Texas, became the first veteran in the United States to receive Qalsody, an FDA-approved medication for SOD1-mutated ALS.
- Qalsody represents the first approved treatment targeting a genetic cause of ALS, specifically for patients with mutations in the superoxide dismutase 1 (SOD1) gene, affecting 1-2% of ALS patients.
- The Houston VA ALS Center's rapid implementation of this cutting-edge therapy demonstrates the VA's commitment to providing veterans with timely access to innovative treatments.
- Veterans face higher ALS risk compared to the general population, making early detection and specialized care crucial for optimal quality of life management.
U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR
- The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence.
- FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020.
- mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.
Tofersen Shows Promise in Phase 1/2 Trial for Inherited Form of ALS
- Biogen's experimental drug tofersen demonstrated safety and successfully lowered levels of disease-causing SOD1 protein in patients with a rare inherited form of ALS in a phase 1/2 clinical trial.
- The antisense oligonucleotide treatment reduced SOD1 protein concentrations by up to 33% in the high-dose group, with some indications it may slow disease progression, prompting advancement to phase 3 trials.
- While targeting only approximately 2% of ALS patients with SOD1 mutations, researchers believe this approach could establish a platform for treating other forms of ALS and neurodegenerative conditions through protein regulation.