Product Approvals and Launches

From respiratory conditions to rare diseases and beyond, here are 5 groundbreaking FDA approvals from last month.

FDA expands selumetinib approval to younger children with NF1-related tumors.

A new FDA-approved blood test for Alzheimer disease could transform diagnosis and treatment accessibility, according to this conversation with Howard Fillit, MD, and Anthony “Nino” Sireci, MD, MSc.

The FDA has approved rilzabrutinib (Wayrilz; Sanofi) as a groundbreaking treatment for immune thrombocytopenia (ITP), enhancing patient outcomes and quality of life.

The FDA has approved an updated COVID-19 booster while limiting access to high-risk individuals, amid ongoing debates on vaccine safety and efficacy.

In the final clip, James D. Chalmers, MBChB, PhD, notes that while the FDA approval of brensocatib is a milestone, questions remain on optimal patient selection, long-term benefits, and further targeting inflammation.

Evolocumab is now indicated for adults who don’t have a prior cardiovascular disease diagnosis.

Brensocatib, the first FDA-approved dipeptidyl peptidase 1 (DPP1) inhibitor for non–cystic fibrosis bronchiectasis, may have broader potential for other neutrophil-driven conditions, according to James D. Chalmers, MBChB, PhD.

James Chalmers, MBChB, PhD, highlights the reassuring safety and tolerability profile of brensocatib in patients with non–cystic fibrosis bronchiectasis.

The approval makes donidalorsen the only treatment in the US for hereditary angioedema that is an RNA-targeted prophylactic.

James D. Chalmers, MBChB, PhD, explains that the ASPEN trial findings show that brensocatib reduces exacerbations, slows lung function decline at higher doses, and offers clinicians a long-awaited evidence-based treatment option.

Today, the FDA issued a complete response letter (CRL) for vatiquinone (PTC Therapeutics), delaying its potential approval as a treatment for Friedreich ataxia amid efficacy concerns.

The FDA approved zopapogene imadenovec, the first therapy for recurrent respiratory papillomatosis, providing an option beyond repeated surgeries to manage the rare condition.

This approval brings the total to 4 of medications approved to treat fibromyalgia; in addition to cyclobenzaprine HCl sublingual tablets (TNX-102 SL; Tonmya; Tonix Pharmaceuticals), there are pregabalin (Lyrica; Viatris), duloxetine (Cymbalta; Eli Lilly), and milnacipran (Savella; AbbVie).

Approval was based on part 1 of the ESSENCE trial, with part 2 results expected in 2029.

Brensocatib (Brinsupri; Insmed) gains FDA approval as the first treatment for bronchiectasis, offering hope to patients with the chronic lung disease.

Christina Poh, MD, of City of Hope National Medical Center, highlights the benefit of tafasitamab in improving progression-free survival for heavily pretreated patients with relapsed or refractory (R/R) follicular lymphoma.

The FDA approved multiple groundbreaking treatments in July, enhancing options across patient populations.

Dordaviprone (Modeyso) is now approved to treat recurrent H3 K27M-mutant diffuse midline glioma, an ultra-rare, aggressive brain tumor primarily affecting children.

There are key steps to take that can ensure equitable access to pegcetacoplan, explained Carla Nester, MD, MSA, FASN, who also highlighted remaining questions on long-term outcomes, safety, and pediatric use.

This approval offers a promising new targeted treatment for patients aged 12 and older with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), according to Carla Nester, MD, MSA, FASN, addressing a critical gap left by broad immunosuppressive therapies.

Following the FDA approval of linvoseltamab (Lynozyfic; Regeneron) for heavily pretreated multiple myeloma, Sundar Jagannath, MBBS, highlights its potential for earlier use, increased accessibility, and greater competition in the B-cell maturation antigen (BCMA) bispecific antibody space.

The newly FDA-approved linvoseltamab (Lynozyfic; Regeneron) may improve access to multiple myeloma treatment by offering an off-the-shelf, outpatient option that can be administered in community settings, according to Sundar Jagannath, MBBS.

The FDA approved pegcetacoplan (EMPAVELI; Apellis Pharmaceuticals) as the first treatment for patients 12 years and older with the rare, severe kidney diseases C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

The FDA’s approval of linvoseltamab for adults with heavily pretreated relapsed/refractory multiple myeloma marks a significant advance, with Sundar Jagannath, MBBS, emphasizing its high response rates, manageable dosing schedule, and reduced treatment burden.

Adult patients with moderate to severe hand eczema with previous treatment failure or in whom topical corticosteroids are contraindicated stand to benefit the most from this approval.

ATLAS trial investigator Guy Young, MD, Children's Hospital Los Angeles and University of Southern California Keck School of Medicine, highlights the current treatment options for patients with hemophilia A or B, with or without inhibitors, since the FDA approval of fitusiran (Qfitlia; Sanofi).

In part 2, Hans Lee, MD, shares practical tips for using linvoseltamab in heavily pretreated multiple myeloma and outlines trials that may expand its future role.

The newly approved linvoseltamab (Lynozyfic; Regeneron) has a manageable safety profile, along with a patient-friendly dosing schedule that includes intravenous administration, step-up dosing, and de-escalation to monthly maintenance.

Many FDA accelerated approvals continue to rely heavily on surrogate endpoints, raising ongoing uncertainty about the true clinical benefits of these therapies.