Data from a Phase 2 clinical study of IONIS-PKK-LRx from Ionis Pharmaceuticals, Inc. meets its primary and secondary endpoints, achieving significant reductions in the number of attacks suffered by patients with HAE compared to placebo. The study demonstrated a mean reduction of 90% in the number of monthly HAE attacks in weeks one to 17 [...]
“With our acquisition of Quellis and concurrent financing, we believe Catabasis is well positioned to advance the development of our lead program, QLS-215, as a differentiated and potential best-in-class new therapy for the chronic treatment of patients affected by HAE to prevent attacks,” says Jill C. Milne, Ph.D., CEO of Catabasis Pharmaceuticals, Inc., at the presentation [...]
Takeda Pharmaceutical Company Limited has submitted a New Drug Application to the Ministry of Health, Labour and Welfare in Japan for lanadelumab subcutaneous injection, a monoclonal antibody therapy for prophylaxis against attacks of HAE. In Japan, it is estimated that between 2,000 and 3,000 people are living with HAE, but only approximately 450 have been [...]
The French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d'Utilisation de cohorte (cohort ATU), or Temporary Authorization for Use, for the use of berotralstat to prevent attacks of HAE in appropriate patients aged 12 and older. This cohort ATU allows patients with HAE in France to receive treatment [...]
Providing an operational update and financial results for the third fiscal quarter ended 31 January 2021, KalVista Pharmaceuticals, Inc. CEO Andrew Crockett says: “We are making excellent progress in our commitment to providing those with HAE a complete set of oral options to manage their disease. The data announced last month for KVD900 as an oral [...]
BioCryst Pharmaceuticals, Inc. has submitted a marketing authorization application (MAA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) seeking approval of Orladeyo (berotralstat) for the prevention of recurrent HAE attacks in HAE patients 12 years and older. The MAA was submitted under the MHRA’s new European Commission Decision Reliance Procedure (ECDRP). On 25 [...]
Pharvaris announces dosing of the first patient in RAPIDe-1, an on-demand Phase 2 study evaluating the efficacy, safety, and pharmacokinetics of PHVS416 in patients with HAE due to C1-Inhibitor Deficiency type 1 and 2. “The initiation of this trial signifies another step towards developing an oral treatment for HAE patients experiencing acute attacks,” says Berndt [...]
Intellia Therapeutics, Inc. reports operational highlights and financial results for the fourth quarter and year ended 31 December 2020. “Intellia’s achievements in 2020 reflect important progress on both our full-spectrum strategy and our mission to deliver curative genome editing treatments for people with severe diseases”, says Intellia President and CEO John Leonard, M.D. “We are [...]
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of Orladeyo (berotralstat) for routine prevention of recurrent attacks of HAE in adult and adolescent patients aged 12 years and older. The European Commission (EC) will review the CHMP recommendation and a final approval decision from the EC [...]
“With two approvals for Orladeyo, our launch in the U.S., and the addition of more than 425 million USD through our May and December financings, 2020 was a transformational year,” Jon Stonehouse, President and CEO says at the presentation of the BioCryst Pharmaceuticals, Inc. financial results for the fourth quarter and full year ended 31 December [...]
