Orphan Drug designation is a status granted to drugs or biologics intended to treat patients with rare conditions, defined as those affecting under 200,000 Americans. According to Pulmonary Hypertension News, the FDA recently given this status to PT001, an investigational treatment for patients with pulmonary arterial hypertension (PAH).

PT001

Developed by pharmaceutical company PulsoSIM Therapeutics (“PulsoSIM”), a subsidiary of VeriSIM Life (“VeriSIM”), PT001 is designed to address multiple PAH pathways. Through VeriSIM, PulsoSIM will gain access to novel technological platforms like BIOiSIM to develop beneficial solutions to PAH and similar conditions.

While data has not yet been released, PulsoSIM suggests that preclinical evaluations of PT001 have shown promise. In the future, the company hopes to move into proof-of-concept studies to further evaluate the safety, efficacy, and tolerability of PT001.

Alongside its Orphan Drug designation, PulsoSIM also earned a variety of benefits. The designation comes alongside tax credits, fee waivers, increased FDA regulatory assistance, and 7 years of marketing exclusivity (if and once PT001 is approved).

Pulmonary Arterial Hypertension (PAH)

Most commonly, BMPR2 gene mutations cause pulmonary arterial hypertension (PAH). Normally, BMPR2 plays a role in cell growth, division, and death. In PAH, these mutations cause the thickening and hardening of pulmonary arteries, resulting in high pulmonary blood pressure. PAH is both chronic and progressive, meaning it can grow worse over time. An estimated 500-1000 new PAH diagnoses are made in the U.S. each year. Additionally, PAH is 2x more likely to affect females than males, especially those between ages 30-60. Outside of gene mutations, sickle cell disease (SCD), liver disease, HIV infection, and illegal drug use may also cause PAH.

In some cases, patients will not experience PAH-related symptoms until the condition has progressed. Once symptoms appear, these can include:

• Fatigue
• Shortness of breath which worsens during exercise or increased activity
• Chest pain
• Swelling of the lower extremities
• Dizziness and/or fainting
• Exercise intolerance
• Heart palpitations
• Chronic cough