Hi,

Designation as "Breakthrough Therapy" is a way to expedite the development and review of drug application for serious or life-threatening conditions. It's not formal drug approval.

Sponsor say that "Ligelizumab is the first treatment to receive FDA Breakthrough Therapy designation in chronic spontaneous urticaria (CSU) in patients with an inadequate response to H1-antihistamines"

Ref:

https://www.novartis.com/news/media-releases/novartis-ligelizumab-qge031-receives-fda-breakthrough-therapy-designation-patients-chronic-spontaneous-urticaria-csu

FDA explain in details what it means:

"Breakthrough therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.

A breakthrough therapy designation conveys all of the fast track program features (see below for more details on fast track designation), more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. Section 902 of FDASIA requires the following actions, as appropriate:

• holding meetings with the sponsor and the review team throughout the development of the drug
• providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the nonclinical and clinical data necessary for approval is as efficient as practicable
• taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment
• assigning a cross-disciplinary project lead for the FDA review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the cross-discipline members of the review team (i.e., clinical, pharmacology-toxicology, chemistry, manufacturing and control, compliance) for coordinated internal interactions and communications with the sponsor through the review division’s Regulatory Health Project Manager
• involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review"

https://www.fda.gov/regulatory-information/food-and-drug-administration-safety-and-innovation-act-fdasia/frequently-asked-questions-breakthrough-therapies

I found only two protocols for Clin. phase publicly available:

https://clinicaltrials.gov/ProvidedDocs/78/NCT03437278/Prot_000.pdf

https://clinicaltrials.gov/ProvidedDocs/18/NCT02649218/Prot_000.pdf

Best regards,

Tomasz