In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare conditions, defined as affecting fewer than 200,000 Americans. As incentives, drug developers whose therapies earn this status also receive fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. According to MPR, the FDA recently granted Orphan Drug status to omadacycline, a therapeutic option for patients with nontuberculous mycobacteria infections, including nontuberculous mycobacterial (NTM) lung disease.

Omadacycline

So what exactly is omadacycline? According to IDStewardship, omadacycline, sold under the brand name Nuzyra, is:

An aminomethylcycline antibiotic that works via inhibition of the 30S ribosomal subunit, which blocks bacterial protein synthesis.

The treatment helps confer protection against, and clear infection from, a variety of bacteria, including strains which have typically been considered as “drug-resistant.” 

In the past, omadacycline was approved for the treatment of community acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI). Now, researchers are evaluating the treatment as an option for patients with NTM lung disease caused by the Mycobacterium abscessus complex (MABc) within a Phase 2 clinical trial. Altogether, an estimated 75 patients will enroll. During this trial, researchers will explore the safety, efficacy, and tolerability of 300mg orally administered omadacycline over a 12-week (3-month) period. 

Nontuberculous Mycobacterial (NTM) Lung Disease

Caused by various types of mycobacteria, nontuberculous mycobacterial (NTM) lung disease is a serious infection which can cause lung damage and even scarring. Nontuberculous mycobacteria are found throughout our environment, often in soil and water. While many people may be exposed to these bacteria, these bacteria do not always cause infection. Rather, those with pre-existing health issues or those who are immunocompromised may develop an infection. Risk factors include having cystic fibrosis (CF), alpha-1 antitrypsin disease (A1AD), bronchiectasis, a weakened immune system, or being post-menopausal. NTM lung disease is not contagious and cannot be transferred between people. While NTM lung disease can affect people of all ages, it most typically affects older individuals. Treatment may take several years. Symptoms and severity vary between patients. However, some common symptoms include:

• Fever
• Night sweats
• Unintended weight loss
• Recurrent respiratory infections
• Appetite loss
• Chest pain
• Fatigue
• Shortness of breath and/or difficulty breathing
• Coughing up blood
• A severe and chronic cough
• Excess mucus production