Novel Therapies May Bring Relief for Hereditary Angioedema Patients

According to a press release, there are at least four companies working on novel therapies that may bring at least some relief for patients suffering from a rare genetic disease called hereditary angioedema (HAE).

Currently, there is no FDA approved drug for acute attacks of hereditary angioedema (HAE). The therapy available today remains at alleviating the symptoms caused by this rare genetic disorder.

Hereditary angioedema (HAE) is a genetic disease that causes swelling of extremities, face and internal organs and can be life-threatening if not treated properly.

Although the disease is rare (it is estimated that there are only 10,000 people with HAE in the United States) it is a serious issue related to the immunological system of the patient. The disease targets the blood vessels and causes what is known as "attacks" of swelling that may last for days. These attacks usually occur in the arms and legs.

However, swelling of the bowel, face and other internal organs have been observed causing severe pain and disfiguration. Swelling of the throat can induce breathing problems and can be potentially fatal if not treated.

In addition, the disease is easily misdiagnosed since other diseases have similar signs and symptoms. This complicates the management of the disease.

Regarding treatments for HAE available only symptoms are treated today since there is no drug approved for this rare disease. Doctors treating HAE patients are limited to provide symptoms relief care such as pain medications for severe abdominal swelling or intubation for asphyxiating problems in cases of throat swelling. Epinephrine, antihistamines, or corticosteroids (commonly used drugs for swelling) do not work with HAE.

Recently treatment with high dose danazol, an anabolic steroid, have been used for preventing HAE attacks but it does not stop them form occurring. Fresh frozen plasma infusions to prevent acute attacks is also available but its use is controversial. Side effects may include allergic reactions and transmission of blood-borne illnesses.

HAE is caused by low levels or bad functioning of a protein called C1-inhibitor. FDA has not approved until today any replacement therapy with C1-inhibitor, which has been available since the 1970's in Europe and in other countries.

According to the press release there are at least four companies conducting research on novel drugs to treat HAE:

*Dyax Corporation (Cambridge, Mass.) is doing Phase III trial with a drug called ecallantide, which blocks the vasodilating chemical responsible for swelling in HAE.

*Jerini AG (Berlin, Germany) recently completed clinical trials of Icatibant

*Lev Pharmaceuticals (New York, N.Y.) has completed clinical trial with C1 Inhibitor in the United States. Its intravenous C-1 replacement therapy has been used safely and effectively in the Netherlands for more than 30 years.

*Pharming Group (Leiden, The Netherlands) is testing a recombinant C1-inhibitor

Let's hope that these therapies are finally and rapidly approved by the FDA.

Sources:

Lev Presents Significant Data from Pivotal Phase III Trial of . Cinryze(TM) in Acute Treatment of Hereditary Angioedema at ACAAI Meeting. URL: http://www.infobolsa.es/v2002/Noticias/noticias_titulares2.asp?Fuente=IBNW&FechaNot=20071111&Clasif=C&Numnot=103588

United States Hereditary Angioedema Association Press release. Four companies conducting clinical trials URL: http://www.haea.org/clinicaltrial.php