HeartWare International ($HTWR) has reached another huge milestone in developing its ventricular assist system for patients with end-stage heart failure, finishing enrollment in a pivotal U.S. clinical study testing the device as a destination therapy. That's more good news for the Framingham, MA and Sydney-based outfit, barely two weeks after gaining the support of an FDA panel of experts that essentially recommended premarket approval to use it for another indication: as a Bridge-to-Transplant device.
HeartWare completed enrollment for ENDURANCE, a pivotal, 450-patient "destination therapy" clinical trial. And it's a testament to how complex device clinical trials can be. Fifty U.S. hospitals took part in the non-inferiority study, and qualifying patients had end-stage heart failure for which standard treatments have failed. Those patients also were ineligible for a heart transplant.
Trial organizers randomly selected patients in a 2-1 ratio to receive either HeartWare's product or an LVAD made by a competitor already approved by the FDA as a destination therapy. The primary endpoint goal here is survival at two years with the original implant device without a disabling stroke. And patients with the HeartWare implant will be followed up to that two-year point, with additional monitoring for up to 5 years after the implant.
Such studies and their long-term collection of data can be important for devices in the approval process. For example, the FDA often requires post-marketing monitoring once it approves a device, and a company's initiative in engineering its pivotal clinical trial for long-term monitoring and collection of data involving safety and effectiveness can be key. (Of course, the device already has its CE Mark approval in Europe.)
On April 25, the FDA's Circulatory System Devices Advisory Committee voted by a large margin that the benefits of HeartWare's ventricular assist system outweighed any risks in using the device as a bridge to heart transplant for patients with end-stage heart failure. Regulators don't have to follow the panel's recommendation but they usually do. HeartWare's device is considered precedent setting, by the way, because it is implanted next to the heart and its use doesn't call for abdominal surgery typically needed for competing products.
- here's the release