Jerini notches up small win in HAE race

For Jerini, it has been a case of the FDA taking with one hand and European regulators giving with the other. Shares in the company hit record lows yesterday, more than halving on the news that the US regulator had slapped it with a non-approvable letter for icatibant, its treatment for hereditary angioedema (HAE), only for them to rise 33% today with the decision by Europe to recommend marketing of the drug.

The roller-coaster ride has left Jerini in pole position to cross the finishing line in getting an approved HAE product on the market. But the more important race to launch in the US, which is turning into a marathon, still leaves none of the current five companies developing products for the rare hereditary disorder with a product in the world’s most lucrative market.

Now nudging ahead is Lev Pharmaceuticals, which despite receiving an approvable letter from the FDA in January for its C1 inhibitor replacement Cinryze, is due before an advisory committee on May 2, and a PDUFA date is expected sometime before mid October.

As the approvable letter mainly related to the manufacturing of the drug and did not require Lev to conduct any additional trials, to which Lev filed its complete response this week, some are still hopeful that the drug could make history and be the first in the US.

Blood plasma

If, however, the FDA decides against Lev, the prize could be snatched by CSL Behring, which in March applied for marketing approval for C1-INH and is optimistically hoping to get its similar human blood plasma product on the market by the end of the year.

The importance of being first to market is due to the relatively small size of the HAE market, with only one in 30,000 people suffering from the disorder that causes recurrent attacks of painful swelling of the face, hand and intestines. The estimated market opportunity is only thought to be $500m.

With such a small pie to split between five companies, and the fact that the first to market will take the lion share of sales, some of the contenders in the race are starting to look beyond HAE to the wider markets their products might be used in.

Yesterday, Dyax, which is phase III trials with its HAE drug DX-88, announced that it had licensed the North American and European rights to the drug for preventing bleeding during heart surgery to Cubist Pharmaceuticals, in deal worth up to $231.5m.

Hereditary angioedema product portfolio Annual WW Sales ($m)   
Market Status (current) Product   Company Pharmacological Class Indication Summary (active)    2008 2012
  Marketed Berinert P  CSL C1 esterase inhibitor   Hereditary angioedema [Marketed] - -
  Filed Firazyr Jerini Bradykinin B2 antagonist Hereditary angioedema [Filed]; Asthma [Phase II]; Liver disorders [Phase II]; Oedema [Pre-clinical] 41 314
  Cinryze      Lev Pharmaceuticals C1 esterase inhibitor  Hereditary angioedema [Filed]; Myocardial infarction, acute (AMI) [Pre-clinical]; General inflammatory disorders [Pre-clinical]          30 280
  Rhucin Pharming C1 esterase inhibitor  Hereditary angioedema [Filed]; Organ rejection [Pre-clinical]; Oedema [Research project] - -
  Phase III DX-88 Dyax Kallikrein inhibitor Hereditary angioedema [Phase III]; Coronary artery bypass graft (CABG) [Phase II] - 121

Secondary indications

The group, which is set to get double digit royalties and co-promotion rights, refused to speculate on the size of the market. In the US about 500,000 procedures that involve placing patients on an artificial heart pump are carried out every year and at present there is no available drug to prevent blood loss in this setting, since the withdrawal of Bayer’s drug Trasylol in November following increased deaths.

Considering that Trasylol had sales of almost $300m before the safety issues started to come to light, this could make the opportunity for Dyax considerably larger than the $121m of sales by 2012 the group is forecast to get for the drug in HAE alone. Today, Bayer announced that it was facing 78 lawsuits in the US relating to Trasylol.

Pharming, which saw its shares more than halve after the EMEA rejected its drug Rhucin in December, has since vowed to focus on getting HAE approval outside Europe, whilst its strategy in the US has still to be determined. It is also focusing its energies on what it sees as the more valuable area of organ transplantation, a market the company estimates is worth $7bn-$8bn. The company has a pre-clinical research programme for the drug in the organ rejection.

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