FDA approves new COPD drug developed by firm in Raleigh. First new treatment in a decade

A new COPD drug, developed by Verona Pharma, was approved by the FDA Wednesday. The drug, ensifentrine, is the first new type of maintenance treatment for COPD in over a decade.

In clinical trials, ensifentrine provided a “compelling benefit to risk,” said David Zaccardelli, president and CEO of Verona Pharma, which is based in London but has its US headquarters in Raleigh. “I think it’d be the fundamental reason ensifentrine gets prescribed across the treatment spectrum.”

Verona Pharma will market ensifentrine under the brand name Ohtuvayre.

What is COPD?

Chronic obstructive pulmonary disease (COPD) makes it harder for your lungs to function, leading to shortness of breath and chronic cough. COPD affects more than 16 million adults in the United States, and is the sixth leading cause of death. There is no cure for COPD, but symptoms can be managed to some extent with medications and lifestyle changes.

Ohtuvayre has two primary functions. First, it is a bronchodilator, opening airways in the lungs to make breathing easier. Second, it is anti-inflammatory, reducing COPD exacerbations or flare-ups, a sudden worsening of symptoms.

Typically, anti-inflammatory action comes from inhaled corticosteroids, which can increase risk for pneumonia.

Ohtuvayre is the first inhaled therapy that achieves the same goal without the same risk.

Current treatments for COPD include one anti-inflammatory medication and two long-acting medications that are also bronchodilators. These treatments are often taken in combination, depending on the severity of the patient’s COPD and which treatments work for them.

“Many, many, many patients come into my office saying that the currently available therapies are not enough,” said Dr. MeiLan Han, chief of the Division of Pulmonary and Critical Care at the University of Michigan and spokesperson for the American Lung Association. Han previously consulted for Verona Pharma but was not involved in the clinical trial.

First new treatment in a decade

There haven’t been any new advancements in COPD maintenance therapies for more than a decade, leaving the patients who still feel symptoms eager for new options. Much of the current work revolves around combining therapies into single products.

Ohtuvayre was found to improve lung function and quality of life for patients. While the clinical trials did not include many patients who experience frequent flare-ups, the drug was found to reduce flare-ups in its participants by 40%.

The clinical trial did not include patients who are on dual therapy with two bronchodilators or triple therapy with two bronchodilators and an anti-inflammatory medication, the treatment standard for patients with severe COPD and frequent flare-ups.

“At least from what I see in my practice, very few [patients], if any, are going to be just on one bronchodilator,” said Dr. Christopher Mosher, a pulmonologist at Duke University whose research centers around rehabilitation and novel COPD treatments.

The clinical trial primarily included patients who were either on one bronchodilator or none.

Han acknowledged that many patients in the trial “were not on all of the standard medications that we would typically use, but said she would probably try prescribing Ohtuvayre to patients on maximal therapy and ones who can’t tolerate current therapies.

Several clinicians told The News & Observer that it’s not yet clear how this drug will improve patient care in the real world, particularly for those experiencing the worst symptoms.

Zaccardelli said the high benefit-to-risk ratio is likely due to the minimal risk. Based on two years of data, he said, the drug’s safety aligned closely with that of a placebo -- it had few if any side effects.

Dr. David Mannino, co-founder and chief medical officer of the COPD Foundation, agreed that there didn’t seem to be “any major safety signals,” noting that “once you get down to a broader population, something may emerge.”

The safety of this drug will let doctors try ensifentrine on patients and see how it affects symptoms without having to worry about the medical risks.

What will the new drug cost?

Christopher Martin, chief commercialization officer of Verona Pharma, said the company considers pricing within the context of COPD costs to the health care system. “There’s about $50 billion in direct and indirect costs associated with COPD,” he said, and “each exacerbation costs the health care system around $25,000 to $30,000 a year.”

The company said Thursday that the drug would be priced at $2,950 per month to wholesalers. and will be available to the public in July.

Martin said ensifentrine will likely be reimbursed under Medicare Part B instead of Part D because it is delivered by a nebulizer, a device that delivers mists of medicine over the course of minutes. Martin said other nebulizer therapies typically have out-of-pocket costs close to $10 under Medicare Part B, and they expect the same for Ohtuvayre.

For patients not covered by Medicare or Medicaid, Martin said the company is taking steps to “ensure that when a patient is prescribed Ohtuvayre, we believe that they have a very good likelihood to get it.” These steps include providing copay assistance and presenting the data to show insurers that the drug will address the needs of COPD patients and the high costs incurred by COPD.