The U.S. Food and Drug Administration agreed to lift a clinical hold on a mid- to late-stage drug to treat cystic fibrosis patients for two different types of lung infections nine months after invoking it.
Insmed’s (NASDAQ CM:INSM) Arikace is designed to treat cystic fibrosis patients who have lung infections from nontuberculous mycobacteria, or NTM, and those with chronic pseudomonas aeruginosa lung infections.
The Monmouth Junction, New Jersey company believes the drug can fill a growing unmet need for cystic fibrosis patients by delivering high, sustained levels of amikacin directly to the lung, potentially providing sustained improvement in lung function and improvement in patient symptoms with a once-a-day dosing regimen, according to a filing with the U.S. Securities and Exchange Commission. If approved, the drug could be the first inhaled antibiotic for cystic fibrosis patients for once-daily administration.
Behavioral Health, Interoperability and eConsent: Meeting the Demands of CMS Final Rule Compliance
In a webinar on April 16 at 1pm ET, Aneesh Chopra will moderate a discussion with executives from DocuSign, Velatura, and behavioral health providers on eConsent, health information exchange and compliance with the CMS Final Rule on interoperability.
The FDA halted patient recruitment for a phase 3 clinical trial for patients with pseudomonas aeruginosa lung infections in August last year after a parallel trial with rats produced a lung tumor in two rats. It had also suspended clinical trial recruitment for patients with NTM lung infections.
As part of the condition for lifting the clinical hold, the U.S. regulator required the company to do a clinical trial that measures inhalation toxicity using dogs. It also revised its clinical trial population of adult patients who have chronic pseudomonas aeruginosa lung infections. An emailed statement from the company said it is continuing discussions with the FDA to finalize additional details of the phase 3 study protocol.
Earlier this year, the FDA lifted a clinical hold on Insmed’s clinical trial for NTM patients after the company agreed to a phase 2 trial to provide proof-of-concept efficacy and safety data. Meanwhile, Insmed has begun enrolling patients in a phase 3 trial of the drug for patients with with pseudomonas aeruginosa lung infections in Europe.
Arikace has received orphan drug designation for pseudomonas aeruginosa patients, but Insmed is awaiting one for NTM patients. It estimates that the global market potential for the two orphan indications together could be as high as $1 billion, according to the SEC filings.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Insmed originally acquired Arikace as part of a $7.8 million deal to acquire the New Jersey-based Transave at the end of December.
