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 and Bristol-Myers Squibb 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.
How much are AstraZeneca and Bristol-Myers depending on Forxiga's U.S. launch?
AstraZeneca is limping following the patent expiration of antipsychotic Seroquel IR last year. The drug's sales fell 77% in the first half of this year, compared to last year's numbers. It was a drop that took Seroquel from blockbuster territory to $226 million for the period. And the loss fueled the company's 10% drop in overall revenues for the half.
Bristol-Myers' U.S. net sales dropped 22% in the second quarter thanks to the 2012 expirations of high blood pressure drug Avapro and blood thinner Plavix. The company lowered its 2013 forecast due to unexpectedly sluggish sales for blood thinner Eliquis -- and for diabetes drug Bydureon.
Bristol and Astra purchased Amylin Pharmaceuticals last year solely for the GLP-1 drugs Byetta and Bydureon. The latter received approval in January 2012, and Bristol-Myers only reported $66 million in sales for the second quarter this year.
A U.S. approval for Forxiga wouldn't take all of the bumps out of the year ahead. But it would inspire greater confidence in the diabetes partnership.
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 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.
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The article 3 PDUFA Dates That Will Kick Off 2014 originally appeared on Fool.com.
Brandy Betz has no position in any stocks mentioned. The Motley Fool recommends BioMarin Pharmaceutical. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.
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