The European Medicines Agency’s review panel announced its support today for three products that will make a big difference to the small drug makers responsible for their development.
In particular, Canada’s Cardiome Pharma and Europe’s Pharming are no doubt allowing themselves to celebrate long awaited positive regulatory endorsements for their respective therapies, vernakalant for atrial fibrillation and Rhucin, which has been renamed Ruconest, for hereditary angioedema (HAE). Meanwhile Archimedes Pharma also saw its new fentanyl formulation PecFent (previously NasalFent) endorsed, a product the privately held company is hoping can make significant inroads into the breakthrough cancer pain market.
Cardiome has travelled a long road to get to this stage, having failed to win over US regulators twice, mainly over safety concerns in a subgroup of patients (Event – At long last Cardiome hoping vernakalant passes regulators, May 17, 2010).
Last year, the FDA asked the company and its partner for the region, Astellas Pharma, to conduct additional trials, which will not report until next year, meaning approval in the US in unlikely to happen before 2012, analysts believe. As such, this European approval is crucial for Cardiome, in terms of regulatory validation for its product and finances; full approval from the European Commission will trigger an estimated $30m milestone for Cardiome from international partner Merck & Co, which will market the drug outside the US.
In Europe the drug will be called Brinavess, in the US Kynapid. Analysts have pencilled in European sales of $42m by 2016, and $162m in the US. As is often the case analysts covering the smaller company are remarkably more bullish, forecasting royalties for Cardiome of $306m in 2016, greater than end user sales.
The drug, generically known as vernakalant, is an intravenous product designed to treat acute atrial fibrillation; the proposed indication is for the rapid conversion of recent onset of atrial fibrillation to sinus rhythm in adults. It works by selectively blocking ion channels in the heart that are known to be active during episodes of atrial fibrillation, and will be the first drug with this mode of action to reach the market.
In early trade today in Canada, shares in the company were 6% higher at C$8.80, tipping its market value over C$500m. The stock touched a 21-month high of C$8.90 last month, suggesting investors were expecting good news.
However, the real endgame for this product is the oral formulation of vernakalant, which Merck partnered in a global deal struck a year ago (Cardiome delivers a deal worth waiting for, April 9, 2009). News is awaited on the start of a phase III trial, which could provide another boost to confidence in the company.
Pharming meanwhile has finally won backing for Ruconest after years of struggling to convince regulators of its safety and efficacy (Event – With a European partner secured Pharming can focus on regulators, April 16, 2010).
First rebuffed back in 2008 the company ran extra trials, pushing its financing to the limit, to prove the drug can effectively treat patients with the inherited inflammatory condition HAE, which causes intermittent and sudden swelling of the soft tissue.
With a marketing partner, Swedish Orphan Biovitrum, already on board the all important launch in Europe can begin as soon as formal approval is received. Still, the drug enters a fairly crowded market for what is a very niche illness. Ruconest, an enzyme-replacement therapy derived from the milk of transgenic rabbits, will compete with a product from CSL called Berinert P, derived from human blood plasma, that has been available for decades, albeit without an official license. And recently Shire launched Firazyr, a small molecule drug administered via an arguably more convenient injection.
As well as fighting for a corner of the market in Europe, the company can now turn its attention to cracking the US, where the first two products to treat HAE recently reached the market (Dyax finally wins US approval for HAE treatment Kalbitor, December 2, 2009).
Although Pharming’s shares rose yesterday, they dropped today on concerns that the company will need to raise more cash at some point, to pay back debt. The stock closed at 23 cents today, down 13%, marking a slight recovery on the 18 cents touched earlier this month but still only a quarter of the company’s value two years ago.
Tipping the balance
And finally, Archimedes won backing for PecFent, a nasal spray formulation of fentanyl.
The opioid analgesic was first sold by Johnson & Johnson as the skin patch Duragesic, which lost patent protection five years ago after several years of generating more than $1bn in sales. Cephalon then took up the baton and enjoyed several years of success after reformulating the product as Fentora, a tablet placed between the cheek and gum, and Actiq, a lozenge on a stick also for absorbtion in the mouth.
Archimedes believes its nasal spray formulation will provide even quicker pain relief and be more suited for patients very sick with chemotherapy, who often have sores in their mouths.
The company, heavily backed by investment house Warburg Pincus, recently raised £65m ($100m) to set up a US sales force; the company already has a presence in Europe (Archimedes fundraising could be start of breakthrough year, March 3, 2010).
With a US decision imminent as well, Archimedes is poised to launch what has the potential to become a significant new product in this market.