pharvaris’ commitment to patients
Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disease that occurs in approximately 1 in 10,000 to 1 in 50,000 people, according to HAEi.
Pharvaris is dedicated to improving the treatment of HAE and the quality of life for patients through our innovative research, discovery, development, and manufacturing of novel therapeutics. The Pharvaris team is purposeful in its quest to advance new alternatives to injected therapies for all sub-types of HAE and other bradykinin B2-receptor-mediated indications.
Pharvaris is currently advancing its wholly-owned clinical-stage program designed to provide patient benefit in HAE.
- The RAPIDe-1 study is a clinical research study for people who have been diagnosed with hereditary angioedema (HAE).
- The main purpose of the study is to find out how effective three different doses of the study drug, PHVS416, are in relieving symptoms associated with HAE attacks.
- Researchers developed the study drug in the form of soft capsules taken by mouth, which are expected to be a more convenient alternative to an injection into a vein or under the skin for resolving HAE attacks.
Pharvaris Expanded Access Policy
Pharvaris is committed to serving patients’ health needs by developing safe and effective therapies and making them available for use as quickly as possible. We believe that controlled clinical trials provide the best path for Pharvaris to generate the safety and efficacy information needed to obtain regulatory approval so that we can make our medicines available to all patients.
Pharvaris always seeks to provide the quickest and broadest access to its medicines, balancing benefit and risk to all patients. We support the principle of expanded-access programs consistent with the best standards for safe and effective use of an investigational therapy, as well as our ability to logistically provide a product. Pharvaris will consider requests for expanded access from qualified physicians, balancing multiple factors.
When evaluating potential use of an investigational therapy, we will consider several factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ guidelines including both patient and scientific perspectives. Eligibility requirements include but are not limited to:
- Sufficient scientific evidence to support both the safety and the efficacy of the compound’s mechanism for this indication
- A serious or life-threatening illness, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
- Sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
- Support that providing the investigational drug will not interfere with clinical trials that could support the investigational drug’s development or regulatory approval for the treatment indication, which could affect future patient access
The evaluation of these, and other, questions may require additional investigation or interaction with the requesting physician, other clinical investigators, or regulatory authorities. Favorable answers alone provide no guarantee that Pharvaris will be able to provide pre-approval access to an experimental medicine.
Requests for access to investigational therapies must be made by a qualified and licensed physician to Pharvaris () and will be evaluated by a core team. Pharvaris anticipates it will acknowledge receipt of such requests within five business days after receipt of an inquiry.
Pharvaris may revise this policy at any time.
Pharvaris values its relationships with patients and patient advocacy groups. We aim to make significant improvements to treatments that are available to patients with HAE. Supporting and supported by patient advocacy groups, we can help achieve this goal when patients have access to a community of peers and educational materials about their symptoms and therapeutic options.
HAE International (HAEi) is the global umbrella organization for the world’s HAE patient groups dedicated to raising awareness of HAE around the world. https://haei.org/