In an August 3rd news release, biopharmaceutical company RELIEF THERAPEUTICS Holding AG (“Relief”) shared that its therapy RLF-100 (aviptadil) for sarcoidosis received Orphan Drug designation from the FDA. This status is granted to drugs or biologics intended to treat patients with rare conditions (under 200,000 Americans) that fill an unmet need or provide benefits over the current standards-of-care. As a benefit, Relief now receives fee waivers, tax credits, increased regulatory assistance, and 7 years market exclusivity upon approval.
According to Relief, RLF-100 is:
a synthetic [inhaled formulation] of Vasoactive Intestinal Peptide (VIP) consisting of 28 amino acids, which was first discovered in 1970. Human VIP is now known to be produced throughout the body and to be primarily concentrated in the lungs [where it] has shown a multimodal mechanism of action: decrease of inflammatory cytokines release leading to prevention of cytokine storm syndrome and viral replication, immunomodulating effect, vasodilating and bronchodilating effects, and prevention of surfactant depletion.
Outside of sarcoidosis, RLF-100 may also be used to treat idiopathic pulmonary fibrosis (IPF), pulmonary arterial hypertension (PAH), sepsis-induced acute respiratory distress syndrome (ARDS), and asthma. When tested in patients with sarcoidosis, RLF-100 was relatively safe and well-tolerated. Additionally, RLF-100 helped relieve symptoms associated with lung inflammation.
Sarcoidosis is a rare inflammatory condition in which abnormal masses (granulomas) form throughout the body. Most often, these granulomas form in the lungs and lymph nodes, though this condition can affect any of the body’s organs. Sarcoidosis occurs due to immune overactivation or an abnormal immune response, though doctors are not sure what prompts this. Normally, our immune system protects us against foreign invaders through inflammation and other bodily processes. When the inflammation does not stop, excess immune cells cause granulomas. In some patients, sarcoidosis may progress to pulmonary sarcoidosis or pulmonary fibrosis, impacting patients’ ability to breathe. However, this condition is not often fatal. In fact, around 50% of patients experience remission within 3 years of receiving a diagnosis. This condition often manifests between ages 20-40, affects females more than males, and disproportionately affects those of African-American heritage.
Symptoms vary based on patient background and affected organs. When symptoms appear, these include:
- Anxiety or depression
- Swollen lymph nodes
- Unintended weight loss
- Joint pain and swelling
- Coughing or wheezing
- Burning, itching, or dry eyes
- Light sensitivity
- Darker or lighter areas of skin
- Lesions on the skin or lumps under the skin
- Chest pain
- Shortness of breath
Learn more about sarcoidosis here.