Blockbuster FDA Decisions In The Next Few Months--FDA Calendar Predictions

FDA's Upcoming Blockbuster Drug Decisions for Q4 2010

October 11, 2010

Again I am predicting the upcoming FDA Calendar for the next upcoming months for the Fourth Quarter.  This is just an initial walkthrough of all the upcoming decisions.  Lets see how well I do.  I am doing pretty well from October's FDA calendar. 

The FDA has some of its biggest decisions of the year looming in the next few months. The first new lupus drug in half a century, a blood-thinner with mega-blockbuster potential, controversial weight drugs and more are all up for regulatory decisions that can move markets and either throw a bucket of cold water or high octane gasoline on stock prices.

In every case, BioPharma Investor has been following the deals and data every step of the way--from early-stage development right through to the NDA and deal-making phases. Now everyone faces the moment of truth, or at least a complete response letter.

The FDA's final decision may be signaled by an expert panel vote, but this is one area where there really is no sure thing. The agency has appeared to be taking a tough stance on safety, which has bedeviled some late-stage therapies. And its complete response letters are always opened with a mix of dread and hope. We've seen plenty of surprising decisions so far this year, and there will likely be more in the months ahead.

I have to do some more research on my Approval Decisions---Here is an preliminary list of FDA Approvals coming in the 4th Quarter of 2010.  This is just an estimated guess off the top of my head.  Some are toss-ups.  The FDA is leaning more on the Complete Response Letter side which isn't necesarily a death sentence as why the drug was not approved and how it might be possible to get approval.  Usually, it requires more Clinical Trials when a company receives a CRL from the FDA.

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FDA Calendar Predictions

1. Benlysta - Human Genome Sciences and GlaxoSmithKline- Full Approval

2. Bydureon - Eli Lilly, Amylin Pharmaceuticals, Alkermes--Full Approval

3. Brilinta - AstraZeneca---Full Approval, although I am nervous about this prediction.  Caution should be noted

4. Pradaxa - Boehringer Ingelheim---Full Approval
Privately Owned Company

5. Qnexa - Vivus--CRL

6. Contrave - Orexigen--CRL

7. Ipilimumab - Bristol-Myers Squibb---Full Approval, however I have not seen the safety data for the Clinical Trials

8. Cladribine - Merck KGaA--CRL over safety concerns

9. Linjeta - Biodel----CRL

10. Afrezza - MannKind--CRL

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I personally like Human Genome Science's Benlysta.  It will be the first treatment for Lupis in a long time.  I think Bydureuon for Diabetes therapies could gain FDA approval.  I think AstraZeneca has a good possibility for FDA approval.  Also, Bristol Myers Squibb with Ipilimumab for Melanoma Cancer Therapy could gain approval.

Benlysta - FDA's Top 10 Blockbuster Decisions

The drug: Benlysta

The partners: Human Genome Sciences and GlaxoSmithKline

PDUFA date: December 9

Market potential: $3 billion-$4 billion plus

By the time the FDA was finished trashing Human Genome Science's troubled hepatitis C drug Zalbin with a complete response letter last week, it would have been hard to find anyone surprised by it. Novartis walked away from the partnership and the drug--along with another $230 million in write-offs--and HGS put the program up on a shelf. But HGS's shares weren't even dented by the decision, as all of its investors refuse to be distracted from the looming FDA ruling on Benlysta.

By the company's count the average forecast on peak sales for Benlysta come in at $4.1 billion. Not everyone is that bullish, but a new first-line therapy for 300,000 U.S. patients and many more around the world could command a jaw-dropping price. And that would translate into the transformation of HGS from a pure developer into a marketing and sales operation with a significant pipeline.

Earlier this year the FDA decided to execute a priority review of Benlysta, and the analysts have given HGS and partner GlaxoSmithKline a solid shot at obtaining an approval on one of the biggest drugs of the season.

Of course, success in biotechnology almost always translates into speculation about a buyout, and HGS has been the subject of much market gossip about a potential GSK tie-up. The confluence of Benlysta buzz and an avid interest in predicting the next big M&A deal has helped boost its market cap into the stratosphere, leaving many analysts scratching their heads over how high it can go.

Bydureon - FDA's Top 10 Blockuster Decisions

Drug: Bydureon

Partners: Eli Lilly, Amylin Pharmaceuticals, Alkermes

PDUFA date: October 22

Peak sales: $1 billion-$6 billion
It's no simple task developing next-gen diabetes drugs; a lesson Roche learned first-hand with its recent taspoglutide debacle. GLP-1 drugs are expected to grab a big share of the growing market, and analysts are now betting that Bydureon--a long-acting version of Byetta that is injected once a week--will be a likely star performer.

Sales estimates are all over the map, ranging from $1 billion to a heady $6 billion, demonstrating once again how hard it is to predict anything in the biopharma world. But there's considerable consensus that the three companies partnered on this program are going to come out well ahead.
Bydureon is a particularly crucial product for Eli Lilly. While its Big Pharma brethren have sallied from their gates to buy out competing drug empires, Lilly has stuck resolutely to its in-house pipeline, with some bad trial bruises to show for its efforts. Lilly badly needs this win to help bolster confidence in CEO John Lechleiter's leadership and the company's future.

Brilinta - FDA's Top 10 Blockuster Decisions

Drug: Brilinta (ticagrelor)

Company: AstraZeneca

PDUFA date: December 16, 2010

Market potential: $2 billion-$4 billion

AstraZeneca needs this win, and the advisory panel was inclined to give it to them. To win European approval in September the pharma company mounted a head-to-head trial with Plavix (2009 sales of $9.5 billion) and won on the data. Now it's looking for an FDA approval of the blood-thinner, and it's mounting a major three-year study to see if Brilinta (ticagrelor) and aspirin can help protect patients most at risk of a heart attack--something Plavix wasn't able to accomplish in a 2006 study.

One of the biggest hang-ups about Brilinta is that in the pivotal trial, Americans didn't respond as well to the therapy as people living outside the U.S., a fact that AstraZeneca attributed to higher overall use of aspirin on this side of the pond. But most of the FDA staffers who poured over the data liked what they saw--and that impressed analysts as AstraZeneca headed into the final stretch.

Pradaxa - FDA's Top 10 Blockbuster Decisions

Drug: Pradaxa

Company: Boehringer Ingelheimsss

PDUFA date: By about October 19

Market potential: Estimates start at $1.3 billion a year

Boehringer Ingelheim won't have long to wait for the FDA's final decision on this blood thinner, which should be just days away. But Pradaxa's blockbuster status is likely to be significantly influenced by a debate over dosing.

Pradaxa earned a unanimous endorsement from the FDA panel. But FDA staffers have been pushing for an approval on the higher, 150 mg dose while dissing the 110 mg dose. That may seem like a small affair compared to an approval versus nonapproval, but Pradaxa faces some big-time competition in Xarelto (J&J) and apixaban (BMS, Pfizer) as the cream of next-generation successors to the standard warfarin.

The winners in this race will be struggling to land a chunk of a market that will swell to anywhere from $10 billion to $20 billion in the next decade, making this one of the top drugs to watch as it plows ahead in its market. There's a high chance of success here, but Boehringer won't be truly happy with half of a loaf. Competitors are angling to prove that they can do the best job, and the stakes couldn't be bigger.

Qnexa - FDA's Top 10 Blockbuster Decisions

Drug: Qnexa

Company: Vivus

PDUFA date: Oct. 28

Peak sales: Who knows?

One of the hottest late-stage development races in biotech is being played out among three public companies--Vivus, Arena and Orexigen--which are laying it all on the line. Unfortunately, they've helped illustrate just why so many Big Pharma companies fear to tread in a development arena where the safety bar is placed very, very high.

Just how high became evident when an expert FDA advisory panel took a close look at some promising one-year safety and efficacy data on Qnexa and then voted 10 to six against a recommendation. There were some big fears that the diet drug could trigger cardiovascular and other side effects which the one-year data wouldn't pick up on. And with the drug designed as an on-going therapy to control weight over an indefinite period, that was a risk they weren't willing to ignore.

Vivus wasn't rolling over, though. Soon after the panel voted the company released solid two-year data with a somewhat improved side effect profile and continued weight-loss well ahead of FDA guidelines for an approval. At a high dose, patients lost an average of 11.4 percent of their weight. That translates to 26 pounds. So far, so good. There's considerable disagreement, though, that the agency will overrule their experts and approve the drug. That could mean a complete response letter conceding approvability but laying out some requirements for continued study. If that means a new trial to examine, say, cardiovascular safety, the drug would be seriously delayed. But not dead.

Read more from TheStreet

Contrave - FDA's Top 10 Blockbuster Decisions

Drug: Contrave

Company: Orexigen and Takeda

PDUFA date: Jan. 31

Peak sales: Who knows?

 To Continue This Story

Arena Pharmaceuticals found itself in a world of hurt after the FDA surprised everyone by announcing its concern for the signs of cancer spotted in animal studies of lorcaserin. A majority of the experts voted against it and the stock tanked as management tried to mollify investors--unsuccessfully--by explaining that they had been right on top of that cancer issue with regulators and that it wasn't relevant to humans. That, evidently, is something that analysts would have liked to have seen as they were evaluating lorcaserin's chances at the FDA.

So what about Orexigen's (OREX) drug Contrave? Biotech scribes have already highlighted their concerns for safety issues, noting the possibility of cardio and psych side effects. Orexigen bet that combining two well known drugs--the antidepressant bupropion and naltrexone--gives it a mountain of safety data to rely on. But analysts note that some of that safety data is outlined on buproprion's black box warning. And there's no way that the agency is going to overlook black box concerns about an obesity drug.

Orexigen shares benefited from Arena's woes, but writers like Forbes influential Matthew Herper note that the obesity market is so enormous, no one is going to be truly competing with each other. Provided, of course, that two of these drugs are approved in the near term.

This has proved one of the toughest challenges in biotech and there's no sign that it's going to get any easier.

Read more on Contrave from the Wall Street Journal

Ipilimumab - FDA's Top 10 Blockbuster Decisions

Drug: Ipilimumab

Company: Bristol-Myers Squibb

PDUFA date: December 25

Peak sales: $1 billion - $1.5 billion

When IMS Health produced the latest bleak assessment of the patent cliff looming on Big Pharma's horizon, analysts wrapped up the review with a few of the brightest drug prospects that could help relieve the sting of lost revenue. Bristol-Myers Squibb's melanoma drug ipilimumab was one of its top five,

BMS landed ipilimumab back in 2009 with its buyout of Medarex, part of an aggressive strategy to remake its pipeline and grab ahold of some late-stage prospects with blockbuster revenue potential. It hasn't been disappointed by the cancer drug. Melanoma is one of the most lethal cancers, so when researchers pulled the veil back on data showing that the therapy significantly improved one- and two-year survival rates, chances of an approval shot up.

Forty-four to 46 percent of patients treated with the drug were alive at one year compared to 25 percent of patients treated with the control arm, BMS reported in June. At two years, 22 to 24 percent of patients treated with ipilimumab were alive compared to 14 percent of patients treated with the control arm.

Ipilimumab is by no means a miracle drug. It's been linked to severe, life-threatening side effects. But analysts have forecast peak sales of $1 billion to $1.5 billion based on its approval for patients with dwindling choices.

Ipilimumab is one of the latest of a string of antibody therapies to make its way through Phase III. Melanoma could be just its first indication as BMS works to get its mojo back in the cancer drug market. And BMS could be on a roll. The blood-clot drug apixaban could well be up for an approval next year, with more blockbuster revenue in the forecast. Bristol-Myers has had its troubles, but with the buyouts still coming, the company has a strategy that has been working well.

Read more on ipilimumab from Bloomberg.

Cladribine - FDA's Top Blockbuster Decisions

Drug: Cladribine

Company: Merck KGaA

PDUFA date: On or about Dec. 5

Market potential: $1.5 billion

At this time last year Merck KGaA was widely billed as a strong contender in the race with Novartis to gain marketing approval for an oral MS drug. But what a difference a few months can make. First the FDA refused to accept the cladribine NDA, saying it still needed work. And then weeks ago European regulators rejected the therapy. That leaves the German company facing a fourth-quarter decision on the would-be blockbuster as Novartis busily rolls out Gilenya at an annual cost of $48,000.

Novartis may have benefited enormously by confronting safety issues raised by Gilenya early on in the trial process. Now that drug is headed to blockbuster status with $1.5 billion-plus in projected sales as it takes on injectables like Rebif and Betaseron. Analysts at Bernstein, meanwhile, are flagging regulatory concerns about Cladribine's long-term immunosuppression with worries about cancer.

The German company has bet a lot on cladribine, which has emerged as its most significant near-term prospect. But a rejection or delay will get Novartis plenty of time to capitalize on a major market shift that will be worth billions of dollars.

Here more on cladribine from the Financial Times, Wall Street Journal and Bloomberg.

Linjeta - FDA's Top 10 Blockbuster Decisions

Drug: Linjeta

Company: Biodel

PDUFA date: Oct. 30

Peak sales: Difficult to project

Discussions about Biodel's (BIOD) fast-acting insulin Linjeta (a new name for what Biodel had called VIAject) often starts with its shortcomings. Trouble in the late-stage Indian arm of the trial left Biodel with far fewer patients to report on than had first been forecast, leading some analysts to question whether the company has the goods to convince regulators that this should be approved now.

Like MannKind, Biodel has had a hard time lining up a major partner to market the product, another sign of some deep-seated worries that Biodel will gain a complete response letter outlining some expensive and extensive new work on the therapy.

But even a delayed approval could be enough to bring some interested parties to the table. Diabetes is a huge market, affecting seven percent of the world's population. And as average weight grows, so does the number of patients. And Linjeta would work for both Type 1 and Type 2 diabetes. By offering a new, fast-acting insulin that can be rapidly absorbed in the blood, Biodel would be tapping into a potentially huge demand.

Would the clarity of a CRL lure a big player like Sanofi or Eli Lilly to the bargaining table? Minyanville thinks so.

Read more on Linjeta from Minyanville.

Afrezza - FDA's top 10 Blockbuster Decisions

Drug: Afrezza

Company: MannKind

PDUFA date: December 29

Peak sales: Difficult to project

Usually the first word you hear when you mention Afrezza is "Exubera," Pfizer's failed attempt at introducing the world to an inhaled insulin product. MannKind founder Alfred Mann has spent a good part of the last few years explaining over and over again that Afrezza's data indicate that his inhaled product is better than injected short-acting insulin-but he still faces a phalanx of market skeptics.

The main advantage of Exubera, though, wasn't that it was more efficacious, but that legions of diabetics would find an inhalable product a much more convenient therapy than something they had to inject. And Pfizer spent millions proving that its theory was wrong.

Adding to the doubt about Afrezza was Mann's insistence on setting a timetable for approval and a partnership that never stayed on track.

And when the FDA's complete response letter for the small device/therapy earlier this year asked for more feedback on clinical utility and the comparability of the treatment used in clinical trials and the one planned for commercial use, investors poured through the exit doors.
Provided MannKind can clear the last big hurdle between Afrezza and the marketplace, though, Mann will get his chance to prove once and for all whether he has a new approach that can revolutionize the way that diabetes is treated--earning blockbuster revenue for the company.
It may be a wild card, but this is one potential blockbuster worth watching.
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