Search results for Breakthrough Therapy Designation

FDA grants breakthrough designation for efimosfermin for MASH

The FDA granted breakthrough therapy designation to efimosfermin — an investigational, once-monthly fibroblast growth factor 21 analogue — for treatment of patients with metabolic...

China Innovative Drug NHWD-870 Receives Breakthrough Therapy Designation, Bringing New Hope of Survival to NUT Carcinoma...

SHAOXING, China, May 8, 2026 /PRNewswire/ -- Recently, the novel oral BET inhibitor NHWD-870 HCI, independently developed by Zhejiang Wenda Pharmaceutical Technology Co., Ltd., off...

<![CDATA[FDA Awards Priority Voucher to Compass for Psychedelic Therapy in Treatment-Resistant Depression]]>

FDA approves Bizengri as first treatment for adults with rare bile duct cancer

The FDA has approved zenocutuzumab-zbco as the first drug for certain patients with a rare and aggressive bile duct cancer.Zenocutuzumab-zbco (Bizengri, Partner Therapeutics) recei...

CMS and FDA Announce New RAPID Program for Medicare Coverage of Breakthrough Medical Devices

On April 23, 2026, the Food and Drug Administration (FDA) and Centers for Medicare & Medicaid Services (CMS) jointly announced the Regulatory Alignment for Predictable and Imme...

‘New era’: FDA grants expanded access to pancreatic cancer therapy

Suneel Kamath, MD, knows too well the sense of helplessness that comes with living through what he calls “the doughnut hole.”After compelling data from a late-stage trial are prese...

RAPID Response: CMS and FDA Team Up to Fast-Track Medicare Coverage for Breakthrough Devices

On April 23, 2026, the Centers for Medicare & Medicaid Services (CMS) and the U.S. Food and Drug Administration (FDA) jointly announced the Regulatory Alignment for Predictable...

A Cancer Driven by Ultra-Rare Mutation Gets Its First FDA-Approved Therapy

Partner Therapeutics’ Bizengri is now FDA approved for treating advanced cases of cholangiocarcinoma driven by NRG1 gene fusions. Partner acquired U.S. rights to this bispecific an...

<![CDATA[FDA Expands Enhertu in Early HER2-Positive Breast Cancer]]>

<![CDATA[FDA Extends Priority Review of Leqembi Iqlik Subcutaneous Injection ]]>

Compass, Usona and Transcend score FDA national priority vouchers amid Trump administration’s psychedelic push

The FDA is awarding national priority vouchers to Compass Pathways, Usona Institute and Transcend Therapeutics for their psychedelic treatments. A third recipient in this batch is...

CMS, FDA announce new program to speed up Medicare coverage of breakthrough medical devices

The Trump administration unveiled a new program to speed up Medicare coverage for breakthrough devices, touting that the new pathway cuts red tape for medical device companies to g...

CorTec receives FDA ‘breakthrough’ designation for brain-computer interface in stroke rehabilitation

CorTec has received “Breakthrough Device Designation” from the US Food and Drug Administration for its Brain Interchange system, a fully implantable brain-computer interface (BCI)...

FDA grants fast track status to 1-time stem cell therapy for hemophilia A

The U.S. Food and Drug Administration (FDA) has granted both fast track and rare pediatric disease designations to Expression Therapeutics’ investigational stem cell therapy for he...

Breaking: FDA Awards Priority Review Vouchers to Otsuka, Compass, and Usona

New Plan Could Speed Medicare Coverage for Innovative Devices

FRIDAY, April 24, 2026 — A new proposal could make it easier for patients to access breakthrough medical devices through Medicare. On Thursday, federal regulators announced a plan...

FDA Issues Recommendations for the Development of Individualized Therapies for Patients With Ultra-Rare Diseases

As discussed previously (“New FDA Guidance Allows Biosimilar Applicants to Use Data From Outside the U.S. To Accelerate Their Approval in the U.S.”), the U.S. Food and Drug Adminis...

FDA fast-tracks the development of psychedelic medications following president’s executive order

The Food and Drug Administration today announced it is accelerating regulatory action on a new class of psychedelic-based therapies, following an April 18 executive order calling t...

<![CDATA[FDA Approves Bizengri for Adults with NRG1 Fusion-Positive Cholangiocarcinoma]]>

Cannabis Extract Gets Breakthrough Therapy Status From FDA

By Pat AnsonA German biopharmaceutical company says its cannabis extract has received a Breakthrough Therapy designation from the FDA, which will speed up its development and revie...

CMS, FDA announce pathway to expedite access to certain medical devices for Medicare beneficiaries

The Centers for Medicare & Medicaid Services and the Food and Drug Administration April 23 announced a new pathway to expedite access to certain FDA-designated Class II and...

CMS and FDA Announce RAPID Coverage Pathway to Accelerate Medicare Coverage for Breakthrough Medical Devices

Key Takeaways: Faster Medicare Coverage. The Centers for Medicare & Medicaid Services (CMS) and the U.S. Food and Drug Administration (FDA) have announced the Regulatory Alig...

Jaguar Health Announces Preliminary Discussion with FDA to Evaluate Breakthrough Therapy Designation for Crofelemer for...

Pediatric patients with Intestinal Failure (IF) due to MVID, an ultrarare congenital disorder with lethal natural history, that has no available therapies and needs lifelong total...

Moderna to Present Phase 1/2 Data on Its Investigational Cancer Antigen Therapy mRNA-4359 as First-Line Therapy in Combi...

The U.S. FDA has granted Fast Track designation for mRNA-4359 in combination with pembrolizumab for the treatment of checkpoint inhibitor refractory unresectable or metastatic mela...