ProQR Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its molecule QR-010 for the treatment of cystic fibrosis (CF) patients who have the deltaF508 mutation in the CFTR gene (the defective gene in CF).
The FDA grants Fast Track status to drugs that are under development and have a serious potential to fill a significant unmet medical need. The intention is to expedite the review process to give patients faster access to the treatment.
QR-010 is a first-in-class RNA (ribonucleic acid)-based oligonucleotide designed to tackle the underlying cause of cystic fibrosis by repairing the mRNA in CF patients with the F508 mutation.
This mutation is a deletion of three nucleotides in the cystic fibrosis transmembrane regulator (CFTR) gene, which leads to the production of a misfolded CFTR protein.
QR-010 is self-administered through a small aerosol device as a mist that is inhaled into the lungs. ProQR developers believe this method allows an optimized delivery of the drug to the primary target organ: the lungs. At the same time, other affected organs also receive the drug through systemic absorption into the blood.
ProQR’s cystic fibrosis candidate has been granted orphan drug status in the U.S. and the European Union, and the project has received funding from a European program called Horizon 2020 to support research and innovation.
The company is currently conducting two global clinical studies for QR-010. One is a proof-of-concept study evaluating QR-010 on the nasal potential difference (NPD). The trial, “Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation,” is a 28-day study with 16 CF participants – eight homozygous (carriers of two copies) for the deltaF508 mutation and eight compound heterozygous (one copy of the mutation plus one other CF-causing mutation) – with the option to enroll 16 more participants.
Top-line data from the first 16 patients is expected to be presented at the 2016 North American Cystic Fibrosis Conference (NACFC) Oct. 27-29 in Orlando, Florida.
The second study is a Phase 1b randomized, double-blind, placebo-controlled, dose-escalation 28-day study. The trial will evaluate QR-010’s safety, efficacy and pharmacokinetics in single and multiple escalation doses of the drug’s inhalation. In total, 64 homozygous (carrying two copies of the deltaF508 mutation) patients will participate in the study.
Exploratory efficacy endpoints include sweat chloride, weight gain, CFQ-R Respiratory Symptom Score, and lung function. Preliminary data for the “Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients” trial is expected to be presented at the 2016 NACFC.
“We are very pleased with the Fast Track designation the FDA granted us for QR-010. It highlights the unmet medical need in cystic fibrosis and the need for innovative and more efficacious medicines for CF. We look forward to working with the FDA to bring QR-010 to patients faster,” ProQR CEO Daniel de Boer said in a press release.
He said ProQR is looking forward to releasing data for the first-in-human trials of QR-010 in cystic fibrosis patients at the NACFC in Orlando.
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