The designation was given based on positive data from the Phase 1 part of the ongoing Phase 1/2 study in which 95% of patients showed a reduction in monthly attack rate post a single dose of NTLA-2002
Intellia Therapeutics headquarters in Massachusetts, US. (Credit: Intellia Therapeutics, Inc.)
US-based Intellia Therapeutics has received Priority Medicine (PRIME) designation from the European Medicines Agency (EMA) for NTLA-2002 to treat hereditary angioedema (HAE).
NTLA-2002 is an in vivo wholly owned investigational CRISPR therapeutic candidate. The first such single dose is designed to deactivate the kallikrein B1 (KLKB1) gene and prevent swelling attacks in HAE patients.
The designation was given based on positive interim data from the Phase 1 part of the ongoing Phase 1/2 clinical study of NTLA-2002 in HAE patients.
As per the results, 95% of patients out of all 10 patients dosed in Phase 1 showed a reduction in monthly attack rate post single dose of NTLA-2002 through the latest follow-up.
The mean duration of follow-up was observed at 9.0 months. In addition, NTLA-2002 was well tolerated at all three dose levels examined, and most side events were mild in severity.
Intellia president and CEO John Leonard said: “We are very pleased the EMA has granted PRIME designation to NTLA-2002. This designation provides valuable regulatory benefits and highlights the potential of our novel in vivo gene editing candidate to address an unmet medical need for people living with hereditary angioedema.
“We look forward to continuing to work closely with the EMA and regulatory agencies around the world to bring this innovative therapy to patients as quickly as possible.”
The global Phase 1/2 study is assessing the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of NTLA-2002 in Type I or Type II HAE adults.
The Phase 1 portion of the study has a goal to detect two dose levels of NTLA-2002 for further assessment in the Phase 2 portion of the study. On the other hand, the Phase 1/2 study will identify the dose of NTLA-2002 for use in future trials.
PRIME is the fourth specialised regulatory designation for NTLA-2002 that the American genome editing company has secured.
The US Food and Drug Administration (FDA) also awarded NTLA-2002 Orphan Drug Designation and RMAT Designation. The UK Medicines and Healthcare products Regulatory Agency (MHRA) has given Innovation Passport to the investigational HAE therapy.