The drug Kalvista obtains the approval of the FDA for the treatment of acute swelling attacks of rare diseases

In rare disease, hereditary edema of angiodema, or HAE, painful swelling attacks can occur anywhere in the body without warning. Medicines are available for the acute treatment of these attacks, but they are all administered by injections or infusions – a heavy dosage method, especially for younger patients. The FDA approved a pharmaceutical pill from Kalvista, making it the first oral therapy to treat acute attacks caused by HAE.

The regulatory decision announced Monday covers the use of medication by patients 12 years and over. Known in development under the name of Sebetrastat, the pill will be marketed under the Ekterly brand. Cambridge, Kalvista, based in Massachusetts, said that his new product, the first in biotechnology, will be launched immediately.

“For the first time, patients have access to oral treatment on demand which offers the possibility of changing how and when the attacks are processed,” said Kalvista CEO, Ben Palleiko, speaking at a conference on Monday morning.

HAE is a hereditary disorder resulting from a genetic deficiency or a dysfunction of the Estérase C1 inhibitor, a protein which controls the proteins associated with swelling and inflammation. The absence of an Estérase inhibitor leads to an uncontrollable activation of the Kallikrein-Kinin system, which in turn leads to the characteristic swelling attacks of HAE. These attacks can occur throughout the body. They can become deadly when affecting a patient’s face and respiratory tract.

The drugs currently available for the treatment of HAE acute attacks include two Estérase C1 inhibitors, Ruconest of Pharming Group and Berinet of CSL Behring. Takeda Pharmaceutical walking a Kallikrein inhibitor, the Kalbitor, and a bradykinine inhibitor, Firazyr, both for the acute treatment of HAE attacks. All of these drugs are administered by needles. Biocrystal pharmaceutical products market an oral inhibitor once a day of Kallikrein, a protein that plays a role in the attacks inflated by Hae. Biocristalon, Orladeyo’s medication is only approved for prevention of HAE attacks in patients aged 12 and over.

Ekterly de Kalvista is an oral inhibitor from Kallikrein with a small molecule. In the phase 3 test which supported the submission of the EKTERLY FDA, the results showed that the drug was faster to obtain a relief of the symptoms, a reduction in the severity of the attacks and an attack resolution compared to a placebo. In an open extension study, Ekterly allowed patients to treat their HAE acute attacks in a median 10 minutes after the start of the attack. These latest results show a relief of the symptoms occurring in a median 1.3 hours for attacks involving larynx and abdomen as well as for attacks pierced in patients who received long -term HAE prophylactic treatment. The most common unwanted reaction in clinical tests was a headache.

Ekterly’s prescribed information allows patients to take a dose, made up of two tablets, at the first sign of a Hae attack. A second dose with two tables can be taken three hours after the first, if the initial dose was inadequate or if the symptoms return or aggravate. Kalvista will put Ekterly available in packages containing two doses or four tablets in total. Kalvista has set a wholesale price of $ 16,720 per dose, which means that a single package costs $ 33,440. In comparison, other brand products available for HAE acute treatment cost between $ 11,000 and $ 17,000 per dose.

“We have evaluated Ekterly competitive with existing brand therapies on the HAE market,” said sales director Nicole Sweeney. “We believe that this price reflects the innovation that we bring to the community and will support wide use.”

In a note sent to investors, Leerink Partners’ analyst Joseph Schwartz said that Ekterly’s own label will open the product to contact a large population of HAE patients.

“With the potential to treat an attack with a pill, which has long been the Holy Grail in Hae, we see Ekterly’s approval while the paradigm moved for the landscape,” said Schwartz. “Ekterly offers an effectiveness of the injectable type, while the practical practical oral route should allow previous treatment and the treatment of all HAE attacks.”

Ekterly is still under regulatory examination in Europe and Japan. Leerink projects the drug will reach around $ 470 million in advanced sales in the United States and Europe for HAE acute treatment. The drug could find additional uses dealing with diseases that benefit from the inhibition of kallikrein. He could also extend his current label in Hae. Kalvista has a pivotal study being evaluated a formulation of ekterly oral disintegration tablets to treat HAE attacks in patients aged 2 to 11.

Competition with Ekterly could come from Pharvaris, who is in pivotal test with the disguctor, an antagonist of the Bradykinine B2 receptors formulated as a capsule once a day. Pharvaris has two phase 3 tests in progress, one for the HAE acute treatment and the other for Hae prophylaxis. The company based in the Netherlands has said that the prolonged release of Deurcicant allows its medication to maintain therapeutic exposure in the body for more than 24 hours, offering the potential of a high level of HAE attacks with a single dose.

Photo by Kalvista Pharmaceuticals