Breakthrough Therapy

FDA Breakthrough Designations Lead to Priority Reviews, Report Finds

3 weeks ago talkbio0Tagged biotechnology innovation, Breakthrough Therapy, CDER, Drug Approval, expedited pathway, Identifier, Infrequent, Neoplasms, Priority Review, regulatory science, United States Food and Drug Administration

FDA; breakthrough therapy designation; priority review; drug approval; expedited pathway; biotechnology innovation; rare diseases; oncology; CDER; regulatory science

Nuvalent Announces FDA Acceptance of New Drug Application for Zidesamtinib in TKI Pre-treated Advanced ROS1-positive NSCLC

3 weeks ago talkbio0Tagged acceptance, ARROS-1 trial, Breakthrough Therapy, Clinical Nurse Specialists, Identifier, New Drug Application, Non-Small Cell Lung Carcinoma, Nuvalent, orphan drug designation, PDUFA, Penetration, pre-treated, Protein-tyrosine kinase inhibitor, ROS1-positive, United States Food and Drug Administration, zidesamtinib

Nuvalent; zidesamtinib; FDA acceptance; New Drug Application (NDA); ROS1-positive NSCLC; tyrosine kinase inhibitor (TKI) pre-treated; ARROS-1 trial; PDUFA date; CNS penetration; breakthrough therapy designation; orphan drug designation

Merck Again Brings Out Big Bucks, Buys Cidara in $9.2B Antiviral Push

4 weeks ago talkbio0Tagged Acquisition, Antiviral Agents, Breakthrough Therapy, CD388, Cidara Therapeutics, Influenza, J&J, Merck, Phase 3, sanofi, United States Food and Drug Administration

Merck; Cidara Therapeutics; CD388; antiviral; acquisition; influenza; phase 3; FDA Breakthrough Therapy; J&J; Sanofi; Keytruda

UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease: Kygevvi for Thymidine Kinase 2 Deficiency (TK2d)

1 month ago talkbio0Tagged Approved, Breakthrough Therapy, doxecitine, doxribtimine, Drugs, Orphan, Infrequent, Kygevvi, Mitochondrial Diseases, Pediatric Disorder, Priority Review, Thymidine kinase 2 deficiency, TK2d, UCB, United States Food and Drug Administration

UCB; FDA approval; Kygevvi; mitochondrial disease; Thymidine Kinase 2 deficiency; TK2d; doxecitine; doxribtimine; rare diseases; Orphan Drug; Breakthrough Therapy; Priority Review; Rare Pediatric Disease

Genmab CEO on the $8B Acquisition of Merus: Strategy Behind the ‘Very Fair’ Deal and Plans to Polish a Rough Diamond

2 months ago talkbio0Tagged Acquisition, Antibodies, Bispecific, Breakthrough Therapy, Cancer of Neck, CEO, expansion, Genmab, Head, Merus, Neoplasms, Perspective, Petosemtamab, pipeline, United States Food and Drug Administration

Genmab; Merus; petosemtamab; acquisition; oncology; head and neck cancer; bispecific antibody; FDA Breakthrough Therapy; pipeline expansion; biotech; $8B deal; CEO perspective

Sanofi’s MS Drug Tolebrutinib Faces FDA Delay Amid Extended Review

3 months ago talkbio0Tagged Breakthrough Therapy, Bruton 's tyrosine kinase inhibitor, Deferred, GEMINI trial, HERCULES trial, Multiple Sclerosis, NDA, nrSPMS, PERSEUS trial, regulatory review, sanofi, Tolebrutinib, United States Food and Drug Administration

Sanofi; tolebrutinib; FDA delay; multiple sclerosis; nrSPMS; Bruton’s tyrosine kinase inhibitor; breakthrough therapy; HERCULES trial; GEMINI trial; PERSEUS trial; NDA; regulatory review

Avidity Touts Functional Improvements for DMD Therapy, Clearing Way to FDA

3 months ago talkbio0Tagged Approved, Avidity, Biosciences, Blackfoot language, Breakthrough Therapy, Clinical Trials, Functional, Improvements, Muscular Dystrophy, Duchenne, skipping, Submission, United States Food and Drug Administration

Avidity Biosciences; del-zota; DMD; Duchenne muscular dystrophy; FDA approval; functional improvements; exon 44 skipping; Breakthrough Therapy; clinical trial; BLA submission

Daiichi and Merck’s Lung Cancer ADC Achieves 48% Response Rate in Pivotal Trial

3 months ago talkbio0Tagged Accelerated, Antibody-Drug Conjugates, Approved, Breakthrough Therapy, CD276 gene, Co., Daiichi, Deruxtecan, Frequency of Responses, IDeate-Lung01, Identifier, ifinatamab, Overall Survival, Progression-Free Survival, Sankyo, Small cell carcinoma of lung, therapeutic autologous dendritic cells, United States Food and Drug Administration

Daiichi Sankyo; Merck & Co.; ifinatamab deruxtecan; I-DXd; B7-H3; antibody-drug conjugate; ADC; small cell lung cancer; SCLC; IDeate-Lung01; objective response rate; accelerated approval; progression-free survival; overall survival; FDA Breakthrough Therapy Designation

Bausch Health to Acquire DURECT and Breakthrough Liver Disease Candidate Larsucosterol for $63M Upfront

4 months ago talkbio0Tagged Acquisition, Bausch Health, Breakthrough Therapy, Dosage Forms, DURECT, epigenetic modulator, Hepatitis, Alcoholic, Larsucosterol, Liver diseases, milestone payments, United States Food and Drug Administration

Bausch Health; DURECT; larsucosterol; liver disease; alcoholic hepatitis; epigenetic modulator; FDA Breakthrough Therapy; acquisition; pharmaceutical; milestone payments

A New Dawn for Psychedelics: Compass Pathways Nears Major Phase 3 Psilocybin Results

7 months ago talkbio0Tagged Breakthrough Therapy, COMP360, Compass Pathways, mental health innovation, Psilocybin, psychedelic therapy, treatment-resistant depression, United States Food and Drug Administration

Compass Pathways; COMP360; psilocybin; treatment-resistant depression; phase 3 trial; psychedelic therapy; FDA Breakthrough Therapy; mental health innovation