FDA Calendar Updates: Repros, Discovery, Acorda, Human Genome Sciences

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 |  Includes: ACOR, GSK, PHUGF, RPRX, WINT
by: Mike Havrilla

Below is a summary of updates to the BioMedReports.com FDA Calendar, which includes a database of over 200 entries. I originally created this calendar to track companies with pending new drug, biological agent, or medical device new product decisions at the FDA. With the launch of BioMedReports.com, the FDA Calendar has expanded to include the following categories: pending new submissions to the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, sBLA filings), pending complete response letter (CRL) re-submissions to the FDA, and pending late-stage pivotal Phase 3 clinical trial results which are designed to support a filing for FDA approval.

On 7/1/09, Repros Therapeutics (NASDAQ:RPRX) announced that an end of Phase 2 meeting is scheduled with the FDA for late September 2009 to discuss a Phase 3 program investigating the use of Proellex in the treatment of endometriosis. The Phase 2 study that RPRX completed earlier this year demonstrated clinically and statistically significant reductions of the three key pain symptoms commonly experienced by women with endometriosis: dysmenorrhea (painful menses), non-menstrual pelvic pain, and dyspareunia (painful intercourse).
Additionally, the reduction of pain was accompanied by a statistically significant reduction in the number of patients requiring pain medication in both doses in this Phase II study compared with placebo. The study showed no efficacy differences between the 25 mg and 50 mg doses. At the upcoming end of Phase II meeting, RPRX anticipates obtaining direction from the FDA on the objectives and endpoints the Agency would like to see used in the pivotal phase 3 efficacy and safety studies in endometriosis. Repros plans to initiate these studies with doses of 12.5 and 25 mg during 4Q09 and 1Q10, in both the U.S. and Europe. RPRX expects to file NDAs for the uterine fibroids indications in the second half of 2010.

On 7/1/09, Discovery Labs (NASDAQ:DSCO) provided an update following the receipt of written minutes from its 6/2/09 meeting with the FDA which followed the Agency's 4/17/09 Complete Response Letter (CRL) for Surfaxin (lucinactant) for the prevention of Respiratory Distress Syndrome (RDS) in premature. At this meeting, DSCO learned that the FDA will now apply a newly-defined standard to determine whether the Company has adequately demonstrated comparability of Surfaxin clinical to commercial drug product. DSCO stated that this new standard represents a significant hurdle for the approval of Surfaxin.

Considering the Agency’s newly-defined standard, DSCO now believes that it is unlikely to satisfy this requirement with existing preclinical comparability data and gain Surfaxin approval in the near term. DSCO stated that the central remaining issue relates to whether data generated using the BAT and preterm lamb model of RDS supports the comparability of Surfaxin clinical to commercial drug product to the FDA's satisfaction. This comparability issue is limited to this NDA for Surfaxin and does not alter the Company’s existing clinical programs or development plans for its pipeline.

DSCO will now focus on maximizing the inherent value of its novel KL4 surfactant and aerosolization platforms and will minimize development risk by leveraging Surfaxin's established proof-of-efficacy in RDS. The two highest priority pipeline programs are Surfaxin LS and Aerosurf and the Company stated that the synthetic nature and formulation flexibility of Discovery Labs' KL4 surfactant platform also supports expansion into a wide range of respiratory disease conditions. DSCO intends to pursue these opportunities through strategic alliances, although there can be no assurance that such alliances can be obtained.

On 7/1/09, Acorda Therapeutics (NASDAQ:ACOR) announced an exclusive deal with Biogen Idec (NASDAQ:BIIB) to develop and commercialize Fampridine-SR in markets outside the U.S. while Acorda will continue to develop and commercialize Fampridine-SR in the U.S. Acorda will receive an upfront payment of $110 million and additional payments of up to $400 million based on the successful achievement of future regulatory and sales milestones along with tiered, double-digit royalty payments on ex-US sales. The FDA is currently reviewing a New Drug Application (NDA) for Fampridine-SR with a Priority Review (six-month) designation and a PDUFA action date of 10/22/09 for a possible FDA decision.
On 4/23/09, ACOR announced the resubmission of its NDA for Fampridine-SR to the FDA as a new therapy being developed to improve walking ability in people with multiple sclerosis (MS). Acorda received a Refuse to File (RTF) letter for the Fampridine-SR NDA on 3/30/09, which cited the need to correct formatting issues with the application and requested additional supporting information before the NDA could be accepted for review. On 5/6/09, the FDA accepted the NDA with a priority review designation and PDUFA decision date of 10/22/09 as there are currently no FDA approved treatments to improve the walking ability of people with MS.
On 6/11/09, Human Genome Sciences (NASDAQ:HGSI) announced Phase 2 continuation data which demonstrated sustained improvement in disease activity and patient response rate through four years of BENLYSTA therapy in combination with standard of care in patients with serologically active systemic lupus erythematosus (SLE). The frequency of disease flares as measured by the SELENA SLEDAI flare index and by BILAG A or B organ domain scores decreased over four years of BENLYSTA therapy and there was no increase in overall adverse events, serious adverse events, malignancies or serious infections over time. HGSI and GlaxoSmithKline (NYSE:GSK) have selected BENLYSTA as the brand name for belimumab (formerly known as LymphoStat-B).
HGS and GSK expect to report the first Phase 3 data for BENLYSTA in July 2009 from the BLISS-52 trial, with results from BLISS-76 anticipated in November 2009. BLISS-52 and BLISS-76 are the largest clinical trials ever conducted in lupus patients. On 7/1/09, HGSI announced that it expects to have top-line results available on 7/20/09 for BLISS-52, the first of two pivotal Phase 3 clinical trials of BENLYSTA in systemic lupus erythematosus (SLE). HGSI will host a conference call to discuss the results on 7/20/09 at 8:15 AM Eastern.
On 7/1/09, Pharming Group [AMS:PHARM] (OTC:PHGUF) announced that it is on track to re-file a request for European Union (EU) marketing approval for its lead product, Rhucin, in September 2009 as the Company confirmed positive results from a final clinical study that included 70 patients in Europe and North America. According to Pharming, none of the Rhucin treated patients experienced a relapse of symptoms in the same attack and there were no clinically relevant safety concerns for the drug. The European Medicines Agency (EMEA) has twice rejected the Company’s application to market Rhucin, which treats hereditary angioedema (HAE), which is a condition that causes acute attacks of painful swelling of the skin, intestine, mouth, and throat. Pharming expects to submit its EU marketing authorization application for Rhucin in September 2009 with a FDA filing expected to follow.
Disclosure: No positions.