Business, energy, technology, markets and global industry news from Business News Today

United States Food and Drug Administration

PharmaDeviceNews United States Food and Drug Administration
Representative image: Cardiac imaging and hypertrophic cardiomyopathy assessment as Cytokinetics reports positive Phase 3 ACACIA-HCM results for aficamten, supporting a planned FDA filing in symptomatic non-obstructive HCM.
Pharma & Biotech

Aficamten clears pivotal Phase 3 test in non-obstructive HCM as Cytokinetics prepares FDA filing

Aficamten hits both Phase 3 ACACIA-HCM endpoints as Cytokinetics prepares an FDA filing. See what the results mean for Myqorzo growth.

Read More
Representative image: Oral preventive therapy for hereditary angioedema as BioCryst Pharmaceuticals expands ORLADEYO into younger children in Japan, strengthening the drug’s global pediatric HAE growth opportunity.
Pharma & Biotech

Japan approves pediatric ORLADEYO as BioCryst Pharmaceuticals expands hereditary angioedema reach

Japan expands ORLADEYO to children aged 2 to under 12 as BioCryst Pharmaceuticals builds its global HAE franchise. See what comes next.

Read More
Representative image: A clinician reviews gastroesophageal cancer imaging and treatment data as Ziihera moves into first-line HER2-positive disease following its latest United States Food and Drug Administration approval, expanding the commercial opportunity for Zymeworks and Jazz Pharmaceuticals.
Pharma & Biotech

Ziihera moves into first-line HER2-positive cancer treatment after pivotal Phase 3 results

FDA expands Ziihera into first-line HER2-positive gastroesophageal cancer. See how the approval could reshape treatment and Zymeworks growth.

Read More
A representative image illustrating Erasca, Inc.’s ERAS-0015 pancreatic cancer program as the FDA grants Fast Track designation following an encouraging 57% preliminary response signal in KRAS-mutant pancreatic cancer and the company prepares for registration-focused development.
Pharma & Biotech

ERAS-0015 gets FDA Fast Track status after 57% response signal in pancreatic cancer

ERAS-0015 wins FDA Fast Track status after a 57% early response signal in KRAS pancreatic cancer as Erasca prepares three registration-focused trials.

Read More
A representative image illustrating Capricor Therapeutics’ deramiocel Duchenne muscular dystrophy program as the FDA extends its review to November 22, 2026, while new HOPE-3 data strengthen the company’s case around preservation of upper limb function.
Pharma & Biotech

Deramiocel gets three more months at FDA as Capricor shifts Duchenne approval case to arm function

FDA extends deramiocel review to November 22 as Capricor submits new HOPE-3 data supporting an upper limb function indication in Duchenne.

Read More
A representative image illustrating Ambros Therapeutics’ neridronate CRPS-1 program as its merger with Werewolf Therapeutics and $150 million financing support the pivotal CRPS-RISE Phase 3 trial and planned FDA filing.
Pharma & Biotech

Neridronate gets $150m runway as Werewolf-Ambros merger funds pivotal CRPS-1 trial

Werewolf and Ambros secure $150m to fund neridronate through its pivotal CRPS-1 Phase 3 readout and planned FDA filing. See what is at stake.

Read More
A representative image illustrating Rein Therapeutics’ LTI-03 idiopathic pulmonary fibrosis program as the FDA grants Fast Track designation and the inhaled therapy advances toward closely watched Phase 2 RENEW data in the second half of 2026.
Pharma & Biotech

LTI-03 gets FDA Fast Track status as Rein heads toward Phase 2 IPF data

FDA grants LTI-03 Fast Track status as Rein advances its 120-patient Phase 2 IPF trial toward a closely watched second-half 2026 readout.

Read More
Revelation Biosciences advances its Gemini acute kidney injury program toward the Phase 2/3 TITAN study after selecting Avance Clinical to support pivotal trial execution and hospital-based patient recruitment. Representative image.
Pharma & Biotech

Revelation Biosciences has its CRO for TITAN. Now Gemini faces the acute kidney injury test that really matters

Revelation Biosciences names Avance Clinical for the Phase 2/3 TITAN study as Gemini approaches its pivotal acute kidney injury test.

Read More
NTx Bio’s RNA manufacturing technology supports Baylor College of Medicine’s production workflow for Diakonos Oncology’s DOC1021 personalized cancer vaccine in a Phase 1/2 refractory melanoma trial, highlighting the manufacturing challenge behind patient-specific cancer immunotherapies. Representative image.
Pharma & Biotech

NTx Bio supports Baylor manufacturing for Diakonos Oncology’s DOC1021 Phase 1/2 melanoma trial

NTx Bio enters the DOC1021 manufacturing workflow as Diakonos tests a personalized dendritic cell therapy in refractory melanoma.

Read More
A representative image illustrating Regeneron Pharmaceuticals’ Pasatru approval as the FDA clears the new fibrodysplasia ossificans progressiva treatment after Phase 3 data showed a 90% reduction in new heterotopic ossification lesions.
Pharma & Biotech

FDA clears monthly Pasatru after Phase 3 data show sharp drop in abnormal bone formation

FDA approves Regeneron’s Pasatru after Phase 3 data showed a 90% reduction in new FOP bone lesions. See what the new therapy means.

Read More