FDA News: Approvals, Adcomms, Complete Response Letters and Guidances

One red lottery winner or rare disease, multi-level personification, power, reverse search, secret sample, special spy, close-up. Company leader

Rare diseases

Through flux and fluidity, FDA keeps coming back to rare disease

Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.

July 8, 2026

6 min read

Heather McKenzie

Manufacturing

Amgen recalls almost 1 million bottles of heart failure drug over quality issue

July 8, 2026

1 min read

Nick Paul Taylor

Podcast

Vertex carries M&A uptick into H2, IPOs and VC raises also grow, FDA on pace despite turmoil

July 8, 2026

1 min read

Jef Akst

Approvals

Vera moves into growing kidney disease space with FDA’s accelerated IgAN nod

July 7, 2026

2 min read

Annalee Armstrong

Opinion

The FDA must put biotech at its center or continue to cede early research to China

July 6, 2026

4 min read

Kendalle Burlin O’Connell

FDA

Despite chaos and churn, FDA decisions hold mostly steady in H1 2026

July 6, 2026

8 min read

Tristan Manalac

Editorial

Biotech’s China problem: An issue of national security or economic value for the US?

July 2, 2026

6 min read

Jef Akst

FEATURED STORIES

3D. rocket soaring. Different rockets fly high and stand out. different from others, concept startup idea, new generation, faster work better

Huntington’s disease

Skyhawk CEO hopes FDA’s new uniQure position will translate to Huntington’s pill

A recent FDA reversal sparked new hope for patients with Huntington’s disease. Flying under the radar, Skyhawk Therapeutics revealed 12-month functional data from a midstage trial of its own candidate showing improvements on a key disease measurement scale.

June 30, 2026

4 min read

Heather McKenzie

Business meeting concept as a group united together as a partnership symbol of strength.

Duchenne muscular dystrophy

Capricor thrown for another loop as FDA sets adcomm for DMD cardiomyopathy therapy

The FDA plans to hold an advisory committee meeting to discuss Capricor Therapeutics’ application for deramiocel, which the agency rejected last July. The news surprised CEO Linda Marbán, who told BioSpace the FDA has not communicated any issues of concern with the company’s resubmitted application.

June 26, 2026

4 min read

Heather McKenzie

Solution problem concept. Man walking on tightrope. Life coaching, transformation idea. Brainstorming in business. Vector illustration

Opinion

The hidden cost of FDA unpredictability

When the variance can’t be modeled, even disciplined biotech investors stop deploying. Here’s the cheapest fix for biotech’s investability problem.

June 26, 2026

6 min read

David Crean

Subscribe to ClinicaSpace

Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday

THE LATEST

Inmazeb is the world's first treatment for Ebola.

FDA

Regeneron’s Inmazeb Becomes First Ebola Treatment Approved by FDA

Regeneron announced that the U.S. Food and Drug Administration approved Inmazeb, a three-antibody cocktail to treat Ebola infections in adults and children.

October 15, 2020

3 min read

Mark Terry

Policy

Biopharma Update on the Novel Coronavirus: October 13

Please check out the biopharma industry coronavirus (COVID-19) stories that are trending for October 13, 2020.

October 13, 2020

4 min read

BioSpace Editorial Staff

Policy

Biopharma Update on the Novel Coronavirus: October 6

Please check out the biopharma industry coronavirus (COVID-19) stories that are trending for October 6, 2020.

October 6, 2020

4 min read

BioSpace Editorial Staff

FDA

LabCorp Receives EUA from FDA for New High-Speed COVID-19 Test

The new high-speed test uses a high-throughput method comprising heat and technology to extract RNA from samples used for COVID-19 RT-PCR molecular testing.

October 2, 2020

3 min read

Brandon May

FDA

Judge Sides with FDA, Jacobus Pharma in Fight Over LEMS Treatment Approval

Catalyst Pharmaceuticals is reeling this morning after a federal judge dismissed the company’s lawsuit against the U.S. Food and Drug Administration regarding the approval of a rival drug for a rare autoimmune disease from New Jersey-based Jacobus Pharmaceutical.

September 30, 2020

2 min read

Alex Keown

FDA

FDA Gives GSK’s Nucala the Greenlight for Hypereosinophilic Syndrome

The new approval makes it the first FDA-approved therapy in over a decade to treat HES and the third indication for the therapy overall.

September 28, 2020

2 min read

Brandon May

FDA

FDA Grants Mereo’s Setrusumab Rare Pediatric Disease Designation

Under that designation, Mereo may qualify for a voucher that can be used for a priority review of a subsequent marketing application for a different product than Setrusumab.

September 24, 2020

2 min read

Mark Terry