The phrase "PDUFA date" is thrown around frequently when discussing biotech and pharma companies. PDUFA stands for the Prescription Drug User Fee Act, which is a law allowing the Food and Drug Administration to charge companies that file new drug applications. The money goes toward expediting the review process that takes six months for drugs with an accelerated approval and 10 months for a standard filing. Adding the appropriate number of months to the date of filing equals the PDUFA date -- or date by which the FDA is expected to make an approval decision.
Take the PDUFA dates as a suggestion rather than an appointment. The FDA is prone to making decisions earlier -- or later. But the date provides investors a general hint as to when a catalyst could impact share prices.
Here's a look at three PDUFA dates that will kick off 2014.
Forxiga tries again
The diabetes partnership of AstraZeneca (AZN +0.00%) and Bristol-Myers Squibb (BMY +0.22%) have a Jan. 11 PDUFA date for SGLT-2 inhibitor Forxiga. It's another try for Forxiga after the companies received a complete response letter requesting more safety data. The delay allowed Johnson and Johnson's Invokana to become the first-in-class drug in the U.S. market. But Forxiga did become the first SGLT-2 approved in Europe.
Decision Resources predicts that the type 2 diabetes market will double to about $50 billion by 2021. Older drug classes such as DPP-4 and GLP-1 will hold on to a large percentage of the market. But SGLT-2s were so highly anticipated because they're insulin-independent and can appear in combo therapies with insulin without the risk of counteraction, which could prove a major selling point for patients.
But wait, there's more...
The Amylin acquisition also included metreleptin, which treats an ultra rare metabolic condition called lipodystrophy that only affects a few thousand patients worldwide. Metreleptin has a PDUFA date of Feb. 27 after the FDA requested an additional three months to review the data.
A potential metreleptin approval ultimately has the same goals as Forxiga: win approval and make the Astra-Bristol partnership appear more sound.
BioMarin's big drug's day
BioMarin (BMRN 1.05%) has four approved products on the market and five in the pipeline. But Vimizim -- formerly called GALNS -- is the project that's held investor attention. The drug treats a rare genetic condition called Morquio syndrome. Positive phase 3 results last year sent shares popping more than 30%. And more catalysts lurk right around the corner.
Vimizim will face a European Medicines Agency committee in the fourth quarter of 2013 for a potential approval recommendation. Then Vimizim is up for U.S. approval with a PDUFA date of Feb. 28. BioMarin is ready for a quick domestic launch if given the go-ahead.
There's currently no approved treatment for Morquio syndrome, which is why Vimizim received priority review status from the FDA. Vimizim provides a chance for BioMarin to own this market.
That's important because BioMarin specializes in orphan drugs and is better off with as many strong approved products as possible. The company can offset the small patient populations by dominating each market it enters and setting high prices for the drugs.
BioMarin's second-quarter loss was narrower than anticipated. And the company has more catalysts ahead including a phase 3 initiation for breast cancer drug BMN 673.
Foolish final thoughts
Out of the three drugs approaching PDUFA dates in the first quarter, Forxiga stands the greatest risk of being turned away. BioMarin would take a greater hit than Astra or Bristol if Vimizim wasn't approved, but that drug has had a less troubled past.
