Why Orphan Drug Designation Offers Several Benefits

By Richard Mason


The U.S. FDA prioritizes developing new treatments for rare diseases with the ability to grant Orphan Drug Designation. Here we discuss the FDA’s Orphan Drug Designation program.

Medical researcher in protective clothing inspecting a test tube

The U.S. FDA prioritizes developing new treatments for rare diseases with the ability to grant Orphan Drug Designation. Here we discuss the FDA’s Orphan Drug Designation program in reference to AbbVie (NYSE: ABBV), Crispr Therapeutics AG (NASDAQ: CRSP), Cytokinetics, Inc. (NASDAQ: CYTK), and Cardiol Therapeutics (NASDAQ: CRDL | TSX: CRDL).

The Orphan Drug Designation program encourages progress in battling rare diseases and conditions. For drug companies carrying out the research and development, the designation brings several incentives, including:

  • Typically obtain high reimbursement levels

  • Exemption from certain FDA fees

  • Potential seven years of market exclusivity after approval


The FDA recently granted Orphan Drug Designation to epcoritamab, a drug developed by Genmab and AbbVie (NYSE: ABBV) to treat follicular lymphoma. AbbVie is responsible for further global commercialization.

Meanwhile, CRISPR Therapeutics AG (NASDAQ: CRSP) has seen several of its gene therapies achieve Orphan Drug Designation from the FDA and the European Medicines Agency.

Cytokinetics, Inc. (NASDAQ: CYTK) was recently awarded Orphan Drug Designation for treating hypertrophic cardiomyopathy (HCM), which leads to cardiac arrest. The company has previously enjoyed the benefits of several orphan drug designations.

Heart failure has remained the leading cause of death globally for the last 20 years, further exacerbated by mounting diabetes, obesity, and high blood pressure cases. This devastating condition comes in several forms but ultimately leads to diminished quality of life and death.

Every year, over 550,000 new cases are diagnosed in the U.S. That’s why Cardiol Therapeutics (NASDAQ: CRDL | TSX: CRDL) is working vigorously to develop treatments to help people live more comfortably with this destructive disease.

Cardiol Therapeutics is focused on researching and developing an anti-fibrotic and anti-inflammatory therapy to treat cardiovascular disease (CVD). Its lead product candidate, CardiolRx™, is a pharmaceutically produced oral formulation being clinically developed for cardiovascular medicine use.

The company currently has one multi-national clinical trial ongoing, with another expected to commence shortly. Furthermore, it has just announced its third FDA IND authorization for its flagship formulation CardiolRx™ in cardiovascular disease.

Altogether, the company is looking to treat acute myocarditis, recurrent pericarditis, and diastolic heart disease.


Cardiol’s latest IND authorization is for a multicenter Phase II open-label pilot study of CardiolRx™ for recurrent pericarditis. This study will run parallel with the company’s multi-national Phase II acute myocarditis trial, which is expected to commence imminently.

Moreover, this means CardiolRx™ is eligible for orphan drug status in two indications, potentially adding significantly more value to the company.

Cardiol’s recurrent pericarditis study will also assess the improvement in objective measures of disease and, during an extension period, evaluate the feasibility of weaning concomitant background therapy, including corticosteroids, while taking CardiolRx™.

Allan L. Klein, MD, FRCP (C), FACC, FAHA, FASE, FESC, Director Center of Pericardial Diseases and Professor of Medicine, Heart and Vascular Institute, Cleveland Clinic, will serve as study Chair and provide leadership throughout the course of the trial.

Cardiol’s study is expected to enroll 25 patients at major clinical centers specializing in pericarditis in the United States. The study protocol has been designed in collaboration with well-established thought leaders in pericardial disease.

The trial’s primary efficacy endpoint is the change, from baseline to 8 weeks, in patient-reported pericarditis pain using an 11-point numeric rating scale (NRS). The NRS is a validated clinical tool used across multiple conditions with acute and chronic pain, including previous studies of recurrent pericarditis. Secondary endpoints include the pain score after 26 weeks of treatment and changes in C-reactive protein (CRP).

With IND authorization now in place, we look forward to ramping up initiation of this important study. We also anticipate benefiting from the clinical trial infrastructure already established for our multi-national acute myocarditis study, which is expected to commence patient enrollment imminently,” commented Dr. Andrew Hamer, Cardiol’s Chief Medical Officer.

Pericarditis refers to inflammation of the pericardium – the membrane or sac surrounding the heart. Symptoms include debilitating chest pain, shortness of breath, and fatigue, which result in physical limitations, reduced quality of life, emergency department visits, and hospitalizations.

Causes of pericarditis can include infection (e.g., tuberculosis), systemic disorders such as autoimmune and inflammatory diseases, cancer, and post-cardiac injury syndromes. Based on time of presentation, acute pericarditis is a symptomatic event lasting less than four to six weeks. The diagnosis is based on meeting two of four criteria: chest pain, pericardial rub; electrocardiogram changes; and new or worsening pericardial swelling. Elevation of inflammatory markers such as CRP, and evidence of pericardial inflammation by an imaging technique (computed tomography scan or cardiac magnetic resonance) may help diagnose and monitor disease activity.

Although generally self-limited and not life-threatening, acute pericarditis is diagnosed in 0.2% of all in-hospital cardiovascular admissions. It is responsible for 5% of emergency room admissions for chest pain in North America and Western Europe.

Recurrent pericarditis is the reappearance of symptoms after a symptom-free period of at least 4–6 weeks following an episode of acute pericarditis. These recurrences appear in 15% to 30% of acute cases and usually within 18 months. Up to 50% of patients with a recurrent episode of pericarditis experience more recurrences.

Cardiol believes there is a significant opportunity to develop a new oral, well-tolerated, and safe therapy for treating recurrent pericarditis to prevent multiple recurrences in the first place, particularly for colchicine-resistant, intolerant, refractory, and contraindicated patients, and steroid-dependent patients.

The U.S. Orphan Drug Designation program was created to provide a drug sponsor with significant incentives, including seven-year marketing exclusivity and exemptions from certain FDA fees, to develop treatments for diseases that affect fewer than 200,000 people in the U.S. Products with Orphan Drug Designation also frequently qualify for accelerated regulatory review. The prevalence of recurrent pericarditis in the U.S. is estimated at 38,000.

AbbVie (NYSE: ABBV) is a biopharma company. It recently awarded its 2022-2023 class of recipients of the AbbVie Immunology Scholarship, which provides financial support to students living with chronic, immune-mediated diseases pursuing higher education in the United States. The scholarship seeks to empower students to pursue a degree and a life not defined by their conditions.

CRISPR Therapeutics AG (NASDAQ: CRSP) is a gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary platform. The company reported in Q1 that it has significant cash on hand to drive a busy year ahead. Crispr is collaborating with Vertex to focus on using CRISPR/Cas9 to discover and develop potential new treatments.

Cytokinetics, Inc. (NASDAQ: CYTK) is a biopharmaceutical company. It focuses on discovering and developing muscle activators as a potential treatment for debilitating diseases. Having secured long-term capital from Royalty Pharma in Q1, a stronger balance sheet should help it accelerate drug development.


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