Alcyone Lifesciences recently obtained Breakthrough Device Designation from the FDA for their novel implantable intrathecal bolus drug delivery catheter and port system, the ThecaFlex DRx System. The system is intended for use in conditions that require prolonged medication administration directly into the cerebrospinal fluid (CSF), such as some cancers and neurodegenerative diseases. Currently, these patients require frequent, repeated spinal tap procedures in order to obtain correct dosing of medication into the central nervous system. Surgical implantation of the Alcyone ThecaFlex DRx system may obviate the need for this treatment regimen, which may involve repeat anesthesia, repeat radiation exposure, and prolonged hospital stays.
Patients with neurodegenerative diseases such as Spinal Muscular Atrophy (SMA), who tend to develop severe scoliosis requiring spinal fusion and instrumentation, and juvenile patients with Huntington’s Disease, who have significant dystonia compared to adult on-set patients, often are not able to receive spinal tap procedures safely or at all. For these patients, the ThecaFlex DRx System potentially provides both an improved quality of life and an alternative life-saving therapy.
This news follows a December 2018 announcement in which Alcyone declared a partnership with pharmaceutical giant Roche to collaborate in the field of intrathecal therapies for severe neurological conditions. Alcyone is currently preparing the ThecaFlex DRx System for an FDA investigational device exemption (IDE) filing to pursue a clinical trial later this year in patients with SMA.
The FDA Breakthrough designation is intented for devices that treat or diagnose life-threatening diseases or irreversible debilitating conditions, for which no approved alternatives currently exist, and offer advantages over existing treatment methods. The FDA also stipulates that the device must represent a “breakthrough” technology. This designation was drafted in 2017 to replace the earlier Expedited Access Pathway (EAP) program, and is intended to “facilitate the development and expedite the review of breakthrough technologies,” according to the FDA.
Alcyone is based out of Lowell, MA, and specializes in “targeted CNS drug delivery platforms.” Alcyone’s technologies also include a precision micro cannula currently in clinical trials for delivery of the DNATrix, Inc. oncolytic virus to treat patients with brain cancer via their public collaboration with the virus maker. The future for the company’s intrathecal drug delivery platforms includes specialized pumps and manual injectors, with patient-specific sensors and features, that will automate drug delivery and are designed to optimize biodistribution of the drug for improved efficacy and outcomes.