Discovery Laboratories Inc. placed its lead drug candidate before regulatory authorities at the FDA, filing a new drug application for Surfaxin to prevent respiratory distress syndrome (RDS) in premature infants.

The Doylestown, Pa.-based company made the submission largely on the basis of supporting data from a pair of Phase III studies of the drug, a peptide-containing, humanized lung surfactant. At the same time, Discovery Labs is preparing an application for European approval, which it plans to file by the middle of this year.

"Surfaxin literally is the first engineered surfactant that mimics human lung surfactant," John Cooper, the company's executive vice president and chief financial officer, told BioWorld Today. "It had extremely successful results in terms of its data when we ran two Phase III trials."

Results of one of the studies, a 1,294-patient pivotal trial, demonstrated Surfaxin's superiority to Exosurf (GlaxoSmithKline plc), a non-protein containing synthetic surfactant. Survanta (Abbott Laboratories), a cow-derived surfactant and the leading surfactant used in the U.S., served as a reference arm in the trial. The second trial was a 252-patient supportive study that demonstrated Surfaxin's non-inferiority to Curosurf (Dey LP), a pig-derived surfactant and the leading surfactant used in Europe.

"Surfaxin, in all of those situations, has a mortality benefit vs. all of those products, as demonstrated in a Kaplan-Meier curve," Cooper said. "There's no doubt in our minds, based upon the data and what we've seen, that once the neonatology and pediatric community has a chance to see the data, Surfaxin should be able to be proven as one of the - if not the - most effective surfactants in the world."

Discovery Labs said investigators would present more detailed results from the Phase III trials at next month's Pediatric Academic Society meeting in San Francisco.

The company is applying for priority review of the application, which could cut the review time down from 12 months to six months. Two months ago, the FDA named the acute RDS treatment the only applicant within the Division of Pulmonary and Allergy Drug Products to be included in its Continuous Marketing Applications Pilot 2 Program, which is designed to evaluate the benefits of more frequent feedback and interactions as a company moves through the various phases of development with fast-track products. (See BioWorld Today, Feb. 10, 2004.)

Discovery Labs has engaged Quintiles Transnational Corp., of Research Triangle Park, N.C., on a contract basis to sell the product in the U.S., should it receive approval. Cooper, who said Discovery Labs would pay a commission to Quintiles, added that the company would work toward launching the product as quickly as possible upon authorization. For European sales, Discovery Labs has aligned with Esteve SA, of Barcelona, Spain, as a licensing partner in that territory.

Cooper noted that the current surfactant market in the U.S. and Western Europe totals about $200 million. But he said the company could grow that market, given an opportunity for premium pricing as well as expanded indications in neonatal intensive care units. Discovery Labs is preparing to begin a Phase II trial of an aerosolized version of the product for respiratory dysfunction in premature infants. The study is expected to begin in the latter half of this year.

"Ultimately, we think there is an opportunity for a surfactant pipeline of products that could approach $1 billion in the neonatal intensive care unit," Cooper said, labeling Surfaxin for RDS as the first for such indications.

The company is conducting additional trials of surfactant products based on the same technology platform. Among them is a Phase II trial for acute respiratory distress syndrome in adults, with data expected in the latter half of this year, as well as Phase II and III trials for meconium aspiration syndrome in full-term infants. Discovery Labs also is preparing to begin a Phase II trial of an aerosolized surfactant for asthma (development name DSC-104).

Late last month, the company raised $24.2 million in gross proceeds through the sale of 2.2 million common shares. (See BioWorld Today, Feb. 10, 2004.)

On Wednesday, its stock (NASDAQ:DSCO) dropped 42 cents to close at $12.02.