FDA Backlog Leaves Biotech Stocks in Limbo

The most important thing coming out of the U.S. Food and Drug Administration these days is what's not coming out of the agency.

I'm talking about on-time delivery of drug approval decisions.

Top brass at the FDA have given drug reviewers permission to slow down decisions on new drugs. The move comes after the FDA has already missed drug approval deadlines because of a lack of resources -- i.e., too few drug reviewers handling too many drug reviews.

The delays have prompted John Jenkins, the FDA's director of the Office of New Drugs, to ease back on drug approval deadlines by giving reviewers the freedom to take more time, if necessary.The RPM Report, a drug industry trade newsletter, broke the story about Jenkins' decision.

"We will still strive to meet our PDUFA goals whenever we can, however, in some cases we may not be able to meet PDUFA goals, and managers have been given the discretion to make those decisions," Jenkins told The RPM Report.

"PDUFA" stands for the Prescription Drug User Fee Act, which is the federal law that sets out the fees drug companies must pay to the FDA to subsidize new drug reviews. In return, the FDA is supposed to meet certain drug approval timelines, which call for a six-month review time for an accelerated drug approval and a 10-month period for a standard review.

"In the short term, our ability to meet PDUFA goals is expected to decrease," Jenkins said in the report. "In some cases we may have to cut back on work by declining requests from sponsors for guidance (e.g., meetings, multiple cycle SPA reviews) and in other cases we may decide to go past the PDUFA goal date for review of an application. In cases where we decide to go past the PDUFA goal date the sponsor will be notified by the division management of that plan."

For investors, the news is clearly troubling. Investing in biotech or drug companies with drugs pending approval is hard enough; now, the endeavor gets even more difficult since drug approval deadlines are going to be fluid.

Cardiome(CRME Quote) and CV Therapeutics(CVTX Quote) have already been hit with FDA delays. The FDA was supposed to act on Cardiome's heart drug IV vernakalant on Jan. 19; the company is still waiting for the FDA to make a decision.

Last week, the FDA informed CV Therapeutics that the review of its cardiac imaging agent regadenoson was taking longer than expected.

In December, I posted a chart of pending drug reviews in front of the FDA this year. I've updated that list below to include all drug reviews now at the FDA with approval dates expected in 2008. The list was compiled with help from BioMedTracker.com and company reports.

Included on the list are also two upcoming FDA advisory panel meetings, one for Lev Pharmaceuticals'(LEVP.OB Quote) drug Cinryze scheduled for May 2, the other for Cephalon(CEPH Quote) and its pain drug Fentora on May 6.

Given the new FDA fluidity when it comes to PDUFA dates, don't be surprised to see some missed deadlines on the list below.

Some highlights from the updated list:

Gilead Sciences'(GILD Quote) aztreonam lysine for cystic fibrosis has an FDA approval decision date of Sept. 16.

AMAG Pharmaceuticals(AMAG Quote) expects to hear from the agency on ferumoxytol, a new intravenous iron treatment for kidney disease patients, on Oct. 19.

Discovery Labs(DSCO Quote) will look for redemption in May when the FDA makes a decision on Surfaxin for the treatment of infantile respiratory distress syndrome. Management mistakes and manufacturing problems have significantly delayed the Surfaxin program, so this may be Discovery's last shot at a turnaround.

Indevus Pharmaceuticals(IDEV Quote) should be able to make a big dent in the growing "manopause" market, with its long-acting testosterone replacement therapy, Nebido. The FDA decision date is June 27.

Expected FDA decision date	Company	Drug	Indication	Status
1/13/2008	Biogen Idec/Elan	Tysabri	Crohn's Disease	Approved
1/17/2008	ZymoGenetics	rThrombin	Hemostasis	Approved
1/18/2008	Johnson & Johnson	TMC125	HIV/AIDS	Approved
1/19/2008	Cardiome Pharma	Vernakalant IV	Acute Atrial fibrillation	Delayed
1/30/2008	Lev Pharmaceuticals	Cinryze	Hereditary angioedema (HAE)	Complete Response Letter
2/2/2008	Abbott Labs	Humira	Psoriasis	Approved
2/23/2008	Genentech	Avastin	Breast Cancer	Approved
3/7/2008	Spectrum Pharmaceuticals	ISO-Vorin	Bone Cancer	Approved
3/14/2008	CV Therapeutics	regadenoson	Heart Imaging Agent	Delayed
04/01/2008-04/24/2008	Amgen	Nplate	Immune Thrombocytopenic Purpura (ITP)
04/01/2008-04/30/2008	Axcan Pharma	Ultrase Capsules	Exocrine Pancreatic Insufficiency
04/01/2008-04/31/2008	Merck & Co.	Cordaptive	Dyslipidemia/Hypercholesterolemia
4/15/2008	Pozen	Trexima	Migraine Headaches
4/30/2008	Progenics Pharmaceuticals	Methylnaltrexone	Opioid Induced Constipation (AMI)
5/1/2008	Discovery Laboratories	Surfaxin	Respiratory Distress Syndrome (RDS)
5/2/2008	Lev Pharmaceuticals	Cinryze	Hereditary angioedema (HAE)	Advisory Panel Meeting
5/6/2008	Cephalon	Fentora	Chronic/breakthrough pain	Advisory Panel Meeting
5/10/2008	Adolor Corporation	Entereg	Postoperative Ileus
06/01/2008-06/30/2008	Eurand	Zentase	Exocrine Pancreatic Insufficiency
6/20/2008	Millennium Pharmaceuticals	Velcade	Multiple Myeloma (First line)
6/26/2008	Eli Lilly & Company	Prasugrel	Acute Coronary Syndrome (ACS)
6/27/2008	Indevus Pharmaceuticals	Nebido	Hypogonadism
7/21/2008	Theravance	Telavancin	Skin and Skin-Structure Infections
7/27/2008	Vanda Pharma/ Titan Pharma	Iloperidone	Schizophrenia
8/1/08-8/31/2008	BioDelivery Sciences Int'l	BEMA Fentanyl	Pain Indications
8/11/2008	Gilead Sciences	Viread	Hepatitis B
8/29/2008	Johnson & Johnson	Paliperidone palmitate	Schizophrenia
9/13/2008	Cephalon	Fentora	Chronic/breakthrough pain
9/16/2008	Gilead Sciences	Aztreonam lysine	Cystic Fibrosis
10/4/2008	Johnson & Johnson	Ustekinumab	Psoriasis
10/19/2008	AMAG Pharmaceuticals	Ferumoxytol	Anemia Due to Chronic Renal Failure
10/31/2008	Cephalon	Treanda	Non-Hodgkin's Lymphoma (NHL)
Source: BioMedTracker, company reports