Early Access

Early Access to Medicine

Harmony Biosciences is dedicated to developing innovative treatments for patients with significant unmet medical needs. To do this, we conduct clinical trials to assess the safety and efficacy of investigational medicines. If proven in clinical trials we can obtain the necessary approvals from regulatory authorities to market these medicines, making them available to be prescribed by healthcare providers. It is also in the best interest of the community of all potential patients for a new medicine to advance through the U.S. Food and Drug Administration (FDA) approval process.

In some circumstances, patients with serious or life-threatening diseases or conditions who do not have access to clinical trials may seek special access to investigational medicines outside of a clinical trial setting. These situations are often referred to as early access, expanded access or compassionate use.

Harmony shares the urgency of patients and families seeking new treatments and understands the interest in accessing investigational medicines before approval by the FDA. We are committed and diligently working to complete the clinical trial process to then seek FDA approval to market our medicines. Currently, the pathway for access to our investigational medicines, prior to regulatory approval, is through a clinical trial. Harmony does not have a special access program outside of a clinical trial for any of our investigational medicines.

We encourage patients and families who are interested in learning more about a Harmony clinical trial to visit our clinical trials page and to talk with their doctor about potential benefits, risks, and feasibility. In addition, you can search www.clinicaltrials.gov for planned, active, recruiting and ongoing clinical trials.

Patients, families, and healthcare providers with questions may email our Medical Information department at medinfo@harmonybiosciences.com.