FDA Grants Rare Pediatric Disease Designation to BPM31510IV for Primary CoQ10 Deficiency
Treatment for primary coenzyme Q10 (CoQ10) deficiency can include high-dose oral CoQ10 supplementation, but not all patients respond to this treatment.
The FDA granted rare pediatric disease designation to BPM31510IV (BPGbio), an investigational treatment for primary coenzyme Q10 (CoQ10) deficiency. This marks the first time a treatment for this disease state received this designation.1
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“BPM31510 has been developed in topical, [intravenous], and oral formulations and is a prime example of the power of our biology-first approach to drug discovery and development, illustrating the powerful predictive capabilities of our NAi Interrogative Biology platform, and the benefit of validating AI-derived predictions through wet lab experiments and in vivo modeling,” Niven R. Narain, PhD, president and CEO of BPGbio, said in a news release. “By using hypothesis-free causal [artificial intelligence] to analyze vast amounts of biological data, we were able to uncover and validate new potential applications BPM31510, including its potential to address mitochondrial diseases like primary CoQ10 deficiency.”1
The deficiency of CoQ10 is classified as a mitochondrial respiratory chain disorder, according to an article in GeneReviews. The principle clinical manifestations include neurological, renal, cardiac, ophthalmologic, hearing loss, and muscular. Individuals often develop seizures, dystonia, spasticity, intellectual disabilities, kidney disease, and retinopathy. It can also cause weakness and exercise intolerance, according to the authors. Further, data on the prognosis of primary CoQ10 deficiency are limited due to a small number of reports to date.2
Currently, there are no clinical practice guidelines for the management of the disease; however, treatment can include high-dose oral CoQ10 supplementation as some individuals respond well to it. Treatment should also be implemented as soon as possible to limit disease progression. Severe neurological and/or renal damage cannot be reversed, but other manifestations can be.2
In addition to this new designation, the drug has also received orphan drug designation for glioblastoma multiforme and pancreatic cancer. The topical form, BPM31510T, also received orphan drug designation for epidermolysis bullosa.1
For glioblastoma multiforme, investigators examined the drug’s potential as a target for glioblastoma multiforme metabolism as a combination with standard-of-care radiotherapy. The early phases of the trial showed that the drug does have a favorable safety profile with primarily hepatotoxicity and coagulopathy. Investigators included patients with glioblastoma multiforme who had no prior therapy, but those with recent hemorrhage, coagulopathy, or who required anticoagulation were excluded. The primary end point included 6-month progression-free survival, and secondary end points included overall survival and safety. There will be approximately 50 patients in the study, with at least 3 sites in the United States.3
"We are honored to receive this designation for BPM31510," Vijay Modur, MD, PhD, senior vice president and Chief Medical Officer at BPGbio, said in a news release. "This milestone brings us one step closer to providing an effective treatment option for children with primary CoQ10 deficiency. We are now preparing to initiate a trial targeting multiple CoQ10 deficiency mutations with the goal of transforming patient lives.”1
