From rxpgnews.com
FDA Issues Approval Letter for a Humanized Surfactant Product for RDS in Premature Babies
By Akanksha, Pharmacology Correspondent
Feb 15, 2005, 07:47
Discovery Laboratories, Inc. today announced the receipt of an Approvable Letter from the U.S. Food and Drug Administration (FDA) for Surfaxin(r) for the prevention of Respiratory Distress Syndrome (RDS) in premature infants.
The Approvable Letter is an official notification that the FDA is prepared to approve the Surfaxin New Drug Application and contains conditions that the applicant must meet prior to obtaining final U.S. marketing approval. The conditions that Discovery must meet primarily involve finalizing labeling and correcting previously reported manufacturing issues.
Most notably, the FDA is not requiring additional preclinical or clinical trials for final approval. The Company anticipates potential approval and commercial launch of the drug to occur in the fourth quarter of 2005 or first quarter of 2006.
It is the first precision-engineered lung Surfactant Replacement Therapy (SRT). It contains a peptide, sinapultide, which is designed to closely mimic the essential properties of human lung surfactant protein B(SP-B).
Surfactants are substances that are produced naturally in the lungs and are essential to the lungs' ability to absorb oxygen and to maintain proper airflow through the respiratory system. Premature babies are born with a lack of natural surfactant in their lungs. Without surfactant, the air sacs in the lungs collapse and are unable to absorb sufficient oxygen resulting in RDS.
Robert J. Capetola, Ph.D., President and Chief Executive Officer of Discovery, commented, "This is a momentous occasion for Discovery Labs and its prospective patients. We are extremely pleased with the approvable designation, which reflects positively on the results achieved in our Phase 3 clinical program. We are indebted to Charlie Cochrane, M.D., Professor and Co-Founder of The Scripps Research Institute, who is the inventor of the Surfaxin technology and continues to be our most influential scientific advisor.
"Surfaxin is the cornerstone of our Surfactant Replacement Therapy pipeline and potentially sets a new standard for the prevention of RDS. Our mission is to advance to market precision-engineered Surfactant Replacement Therapies with the promise to revolutionize the treatment of respiratory diseases prevalent in the neonatal intensive care unit, critical care, and hospital settings."
RDS is a life-threatening and costly breathing disorder that strikes tens of thousands of premature infants in the United States each year, with a global at risk population in excess of 500,000 infants. Approximately 75,000 infants are treated with surfactants in the United States annually. Current surfactant treatment options are limited to animal-derived surfactants harvested from bovine (cow) and porcine (pig) sources.
The humanized surfactant product candidates are based on the proprietary peptide KL4 (also known as sinapultide) designed to precisely mimic the essential attributes of human surfactant protein B (SP-B), the protein in natural pulmonary surfactant known to be the most important surfactant protein for surface tension lowering and promoting oxygen exchange. Chemically, KL4 consists of 21 amino acid residues where �K� is the amino acid lysine and �L� is the amino acid leucine. KL4-surfactant is an aqueous suspension consisting of KL4, the lipids DPPC and palmitoyl-oleoyl phosphatidylglycerol (POPG), plus the fatty acid, palmitic acid (PA).
Data from Discovery's pivotal, multinational SELECT study demonstrates that the humanized surfactant product was significantly more effective in the prevention of RDS and improved survival and other outcomes versus comparator surfactants. The SELECT and STAR (a supportive Phase 3 study) trials, as well as a pooled Phase 3 analysis, have been presented at several international medical meetings and will be published in a leading medical journal in the second quarter of 2005.
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