Full Press Release Details
Labs Provides Expanded Update Regarding its Program for Surfaxin U.S.
Marketing Authorization
to Hold Conference Call to Detail Progress Towards Surfaxin Complete
PA - February 1, 2011 - Discovery Laboratories, Inc. (Nasdaq: DSCO) is providing an
expanded update regarding ongoing efforts to file a Complete Response intended
to gain U.S. Food and Drug Administration (FDA) marketing authorization for
for the prevention of respiratory distress syndrome (RDS) in premature
infants. On January 10, 2011, Discovery Labs issued a press release
reporting that the FDA had recently provided detailed, written direction in
response to an earlier submitted proposal by Discovery Labs relating to its
ongoing comprehensive preclinical program intended to gain Surfaxin
approval. Throughout the conduct of the program, Discovery Labs has
been interacting with the FDA and incorporating the FDA's guidance into its
efforts to complete the program and file the Surfaxin Complete
Response. The filing of the Complete Response is currently targeted
for early third quarter 2011 and anticipated to lead to a potential approval
early in the first quarter 2012.
Company will host a conference call this morning at 10:00 AM EST to provide an
update regarding certain activities relating to the Surfaxin Complete Response.
is 866-332-5218 (additional call-in information below).
approved, Surfaxin would become the first synthetic, peptide-containing
surfactant for commercial use in neonatal medicine. The safety and
efficacy of Surfaxin for neonatal RDS has previously been demonstrated in a
large, multinational Phase 3 clinical program.
for Neonatal RDS - Status of
Comprehensive Preclinical Program for Filing Complete Response to Gain
FDA Marketing Authorization
Labs believes that a key remaining step to potentially gain FDA marketing
approval for Surfaxin is to satisfy the FDA as to the final validation of the
fetal rabbit biological activity test (BAT). The BAT is an important
quality control release and stability test for Surfaxin. Final BAT
validation is intended to satisfy the FDA with respect to the ability of the BAT
to adequately reflect the biological activity of Surfaxin throughout its shelf
life and to discriminate biologically active from inactive Surfaxin drug
product. Discovery Labs has been conducting a comprehensive
preclinical program with regard to this key remaining step. The BAT
has undergone a number of method refinements intended to optimize its
performance and reduce assay variability. The optimized BAT has
subsequently been used to generate data to support BAT validation as well as to
demonstrate concordance between the BAT and the well-established preterm lamb
model of RDS by performing a series of prospectively-designed, side-by-side
preclinical studies (i.e., concordance studies). Data from the
preterm lamb model and BAT concordance studies are intended to support final BAT
validation and to demonstrate comparability of drug product used in the Phase 3
clinical program with Surfaxin drug product to be manufactured for commercial
Labs has been interacting with the FDA in an effort to ensure that the
comprehensive preclinical program (and the Surfaxin Complete Response) will
ultimately satisfy the FDA as to the final validation of the BAT. The
FDA's most recent communication clearly indicates that several aspects of
Discovery Labs' proposed approach to the BAT validation are reasonable and
provides detailed, written direction regarding certain components of Discovery
Labs' comprehensive pre-clinical program. Discovery Labs believes
that it can incorporate the FDA's direction into its ongoing efforts to complete
the comprehensive preclinical program and be in a position to file a Surfaxin
Complete Response by early third quarter 2011, which, after an anticipated
six-month FDA review cycle, could lead to potential Surfaxin approval early in
the first quarter 2012.
BAT Optimization and Final
Validation: Before undertaking steps to optimize the BAT,
Discovery Labs submitted a proposal to FDA outlining several specific method
refinements intended to improve BAT performance and reduce assay
variability. After taking into account the FDA's response, Discovery
Labs incorporated the proposed method refinements into the BAT and conducted
testing that demonstrates that all pre-specified acceptance criteria were met
and that BAT optimization has resulted in a greater-than-40% reduction in assay
variability relative to the BAT methodology that was employed prior to
BAT optimization, at the FDA's suggestion, Discovery Labs submitted an
additional proposal regarding specific and detailed aspects of final BAT
validation. With respect to certain technical criteria relating to
final BAT validation, the recent communication directs Discovery Labs to
increase the sample size of specified data sets by testing additional Surfaxin
batches. Discovery Labs currently has data from several batches that
have previously been manufactured and analyzed. To be responsive to
FDA's direction, Discovery Labs has initiated and anticipates completing
manufacture of additional Surfaxin batches in the first quarter of this
year. The additional batches will be tested using the optimized BAT
and the additional data will be incorporated into the Complete
Concordance Studies -
Throughout the conduct of the comprehensive preclinical program, Discovery Labs
has interacted with the FDA on certain aspects of the concordance
studies. Multiple Surfaxin batches have been used to assess the
biological activity of Surfaxin via measurement of respiratory compliance at
various time points across the proposed shelf-life in both the preterm lamb
model and the optimized BAT. The FDA has previously indicated that,
to gain Surfaxin approval, data generated in the concordance studies must
demonstrate the same relative changes in biological activity over time in both
models using both regression analysis as well as a point-to-point
comparison. Discovery Labs has evaluated the concordance data
generated to date by these models using both regression analysis and
point-to-point comparisons. Discovery Labs believes that these data
demonstrate the same relative changes in biological activity over time in both
the optimized BAT and the preterm lamb models and support the comparability of
drug product used in the phase 3 clinical program and Surfaxin drug product to
be manufactured for commercial use.
recent proposal submitted by Discovery Labs to the FDA also requested
clarification with respect to certain limited technical aspects of the
concordance study. The FDA's response includes detailed direction
with respect to generating limited additional confirmatory data through further
concordance testing. Discovery Labs plans to generate these
additional data for inclusion in the Complete Response, which data must be