Drugs and Medications Approvals and Updates
FDA Approves Palsonify: First Once-Daily Pill for Acromegaly in Adults
- Inluriyo: FDA Approves New Once-Daily Pill for ESR1-Mutated Advanced Breast Cancer in Patients Previously on Hormone Therapy
September 26, 2025 — The FDA approves Inluriyo (imlunestrant), a new oral treatment for people whose locally advanced or metastatic breast cancer is ER-positive, HER2-negative, and has a faulty ESR1 gene.
- Tremfya: First Fully Under-the-Skin IL-23 Inhibitor Shot Approved for Ulcerative Colitis and Crohn’s Disease
September 23, 2025 — The FDA has approved an under-the-skin injectable version of Tremfya for adults with moderate-to-severe ulcerative colitis (UC) for starting as well as continuing the treatment without the need for intravenous (IV) infusions.
- Keytruda Qlex: FDA Approves New Subcutaneous Immune Therapy Shot for Treating a Wide Range of Advanced Cancers
September 22, 2025 — The FDA has approved Keytruda Qlex, a new form of pembrolizumab given as an injection under the skin that helps your immune system find and attack cancer cells.
- Forzinity: New Once-Daily Shot Gets Accelerated FDA Nod for Boosting Muscle Strength in Barth Syndrome
September 22, 2025 — Forzinity (elamipretide) is the first medicine to receive accelerated approval from the FDA for improving muscle strength in people with Barth syndrome who weigh at least 66 pounds (30 kilograms).
- FDA Approves Enbumyst: First and Only Nasal Spray Diuretic for Edema From Heart, Liver, and Kidney Diseases
September 19, 2025 — Enbumyst is a new nasal spray form of bumetanide (first approved by the FDA in 1983). It is a type of medicine called a loop diuretic (also known as a “water pill”), which helps your body get rid of extra fluid by making you urinate more.
- Opzelura: FDA Expands Use of Steroid-Free Skin Cream for Young Children With Eczema
September 19, 2025 — The FDA has now approved this cream for children as young as 2 years old with mild-to-moderate atopic dermatitis (also known as eczema) in whom other prescription creams don’t work well or aren’t suitable for certain reasons.
- Vyjuvek Label Update: FDA Removes Lower Age Limit
September 17, 2025 — A recent FDA label update now allows Vyjuvek to be used from birth.
- Ocaliva Pulled From U.S. Market for Liver Disease
September 12, 2025 — Intercept Pharmaceuticals announced the voluntary withdrawal of Ocaliva from the U.S. market following an FDA request.
- Inlexzo: A New Bladder Cancer Treatment That Delivers Medicine Directly in the Bladder
September 10, 2025 — Inlexzo is a new treatment for a type of bladder cancer in adults that hasn’t spread to the muscles and doesn’t respond to Bacillus Calmette-Guérin (BCG) therapy.
- FDA Expands Approved Use of Vonvendi
September 8, 2025 — Vonvendi is a lab-made version of von Willebrand factor (VWF), a protein your body needs for blood to clot.
- FDA Approves At-Home Once-Weekly Leqembi Injection as Maintenance Therapy for Early Alzheimer's Disease
September 3, 2025 — The FDA has approved Leqembi (lecanemab-irmb) as a once-weekly shot that can be taken at home for people to continue treatment after completing 18 months of IV therapy. It can also be taken as a once-monthly IV infusion at a clinic for continuing therapy.
- Wayrilz: A New Twice-Daily Pill for Chronic Immune Thrombocytopenia Unresponsive to Other Therapies
September 2, 2025 — Wayrilz (rilzabrutinib) is a new oral medicine for adults with a long-lasting blood clotting disorder known as chronic immune thrombocytopenia (ITP) who haven’t had success with other treatments. Find out more about it.
- FDA Expands Repatha Approval for Adults at Risk of Heart Problems Due to High Cholesterol and Approves Use as Standalone Therapy in Adults and Children with Homozygous Familial Hypercholesterolemia
August 27, 2025 — Repatha can now be used in adults with poorly controlled LDL who haven’t been diagnosed with heart disease but are at high risk for serious heart problems such as stroke, heart attack, and death.
- Dawnzera: First RNA-Targeted Therapy to Prevent Hereditary Angioedema Attacks
August 22, 2025 — Dawnzera (donidalorsen) is a new medicine approved to help prevent attacks of a rare genetic swelling disorder known as hereditary angioedema (HAE) in people aged 12 or older.
- FDA Approves Wegovy as First Once-Weekly Injection for Obesity-Linked Liver Disease
August 19, 2025 — The FDA has approved Wegovy (semaglutide) to treat a liver condition called metabolic dysfunction-associated steatohepatitis (MASH) in adults whose livers have significant scar tissue but don't yet have severe liver damage (cirrhosis).
- FDA Approves New Non-Opioid, Once-Daily Bedtime Tablet for Fibromyalgia Pain and Sleep Disturbance
August 18, 2025 — Tonmya (cyclobenzaprine) is a non-opioid medicine approved by the FDA to treat fibromyalgia in adults.
- Papzimeos: First Approved Immunotherapy for Recurrent Respiratory Papillomatosis in Adults
August 15, 2025 — Papzimeos (zopapogene imadenovec-drba) is a new type of immunotherapy approved to treat adults with recurrent respiratory papillomatosis (RRP).
- Brinsupri: First Once-Daily Tablet for Non-Cystic Fibrosis Bronchiectasis
August 13, 2025 — Brinsupri (brensocatib) is a new oral medicine approved to treat a long-term lung disease known as non-cystic fibrosis bronchiectasis (NCFB) in adults and children ages 12 or older.
- Hernexeos (Zongertinib) Approved as the First Oral Targeted Treatment for HER2-Mutant Advanced Lung Cancer
August 11, 2025 — Hernexeos (zongertinib) is a new pill for adults with a type of advanced lung cancer called non-small-cell lung cancer (NSCLC) that has a HER2 gene mutation that causes the cancer to grow and spread faster, especially to the brain.
- Modeyso (Dordaviprone) as First Treatment for Recurrent H3 K27M-Mutant Diffuse Midline Glioma
August 7, 2025 — Modeyso (dordaviprone) is a new oral medicine that treats a rare and aggressive brain tumor, called diffuse midline glioma (DMG), that has a tumor mutation known to promote tumor growth, called an H3 K27M mutation.
- Ajovy Approved as a First-of-Its-Kind Shot for Preventing Migraine in Children and Teens
August 6, 2025 — Ajovy (fremanezumab) is a medicine approved to help prevent migraines. It is a lab-made monoclonal antibody that blocks a protein called calcitonin gene-related peptide.
- Leqvio: New Injectable Option for Lowering Bad Cholesterol Without Statins
August 4, 2025 — Leqvio (inclisiran) is a prescription injection that helps lower levels of low-density lipoprotein cholesterol (LDL), often called “bad” cholesterol, which is a major risk factor for heart disease, heart attack, and stroke.
- Vizz: New Daily Eye Drop to Improve Age-Related Blurry Near Vision
August 4, 2025 — Vizz (aceclidine ophthalmic solution) is a new prescription eye drop approved to treat presbyopia, a common condition that makes it harder to see things up close as you get older. It's the first eye drop in the U.S. to use aceclidine.
- Alhemo: A New Daily Injection to Help Prevent Bleeding in Hemophilia A and B
August 4, 2025 — Alhemo (concizumab-mtci) is a medicine approved to help prevent bleeding in people aged 12 or older with hemophilia A or B who don’t have inhibitors to certain blood clotting proteins.
- Empaveli: A New Treatment Option for Rare Immune-Related Kidney Diseases
July 30, 2025 — Empaveli (pegcetacoplan) is now approved as the first targeted medicine for adults and children aged 12 years or older with two rare and serious kidney diseases: complement component 3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC‑MPGN).
- Skytrofa Approved as Once-Weekly Option for Adults With Growth Hormone Deficiency
July 29, 2025 — Skytrofa (lonapegsomatropin-tcgd), a long-acting version of human growth hormone (hGH), is now approved for adults with GHD.
- New Oral Treatment for Adults and Children With Phenylketonuria: Sephience
July 29, 2025 — Sephience (sepiapterin) is a new oral medication approved by the FDA to treat a rare inherited condition known as phenylketonuria (PKU) in adults and children aged 1 month or older.
- New Oral Option for Children With Low Platelet Counts: Doptelet
July 28, 2025 — Doptelet (avatrombopag) is a prescription medicine that helps the body make more platelets (cells that help stop bleeding). First approved in 2018 for adults, it is now also approved by the FDA for children aged 1 year or older with chronic immune thrombocytopenia (ITP) who haven’t gotten good responses from earlier treatments.
- FDA OKs New Steroid-Free Skin Cream for Hand Eczema
July 24, 2025 — The FDA has approved a new treatment, sold under the name Anzupgo and containing a drug called delgocitinib, as the first skin cream for adults with long-term or chronic hand eczema (CHE), which affects about 1 in every 10 people globally.
- FDA OKs Moderna’s COVID Shot for Kids, With Limits
July 11, 2025 — The FDA has given full approval to Moderna's COVID-19 shot, Spikevax, allowing it to be used in children 6 months to 11 years old, with some limits on who can get it.
- FDA Approves First Oral Treatment for Rare Swelling Disorder
July 8, 2025 — The FDA has approved sebetralstat (sold as Ekterly), the first-ever pill for people 12 and older with hereditary angioedema, a rare genetic disorder that causes sudden and painful swelling that can sometimes be life-threatening.
- FDA OKs First-of-Its-Kind Lung Cancer Pill
July 7, 2025 — The FDA has granted quick approval to sunvozertinib (sold as Zegfrovy) a first-of-its-kind pill for a type of lung cancer known as non-small-cell lung cancer (NSCLC).
- FDA Greenlights New Treatment for Advanced Blood Cancer
July 3, 2025 — A new treatment has received a quick federal nod for a common blood cancer, which could affect more than 36,000 people and result in around 12,000 deaths in the U.S. this year alone.
- FDA Approves First Twice-Yearly Shot to Prevent HIV
June 19, 2025 — The FDA has approved the first twice-yearly shot to reduce the risk of HIV infections.
- FDA OKs New Monthly Treatment for Rare Swelling Disorder
June 18, 2025 — The FDA has approved a first-of-its-kind treatment for a rare genetic condition that causes frequent, unpredictable, and painful swelling in different parts of the body.
- FDA OKs New Next-Gen Pill for Rare Lung Cancer
June 12, 2025 — The FDA has approved an oral pill called taletrectinib (Ibtrozi) to treat people who have a rare type of non-small-cell lung cancer (NSCLC) that is linked to a faulty ROS1 gene and has spread or worsened.
- FDA Approves New 3-in-1 Combo Pill for High Blood Pressure
June 11, 2025 — The FDA has approved a new pill called Widaplik, which combines three drugs – telmisartan, amlodipine, and indapamide – to treat adults who have high blood pressure.
- FDA OKs New Shot to Protect Babies Against RSV
June 10, 2025 — The FDA has approved clesrovimab -- sold as Enflonsia -- a new shot to prevent respiratory syncytial virus (RSV) infection in babies during their first RSV season, which typically lasts from fall through spring.
- FDA Approves Moderna’s New COVID Vaccine With Restricted Use
June 2, 2025 — The FDA has approved Moderna's new COVID-19 shot, an mRNA vaccine called mNexspike, with some limits on who can get it.
- FDA Approves New Treatment for Dry Eye Disease
May 30, 2025 — The FDA has approved Tryptyr (acoltremon) as the first TRPM8-targeting eye drop for dry eye disease, stimulating natural tear production as early as day one.
- FDA OKs First Meningococcal Vaccine for Infants
May 30, 2025 — The FDA has approved MenQuadfi as the first-ever meningococcal vaccine for babies as young as 6 weeks old. It protects against invasive meningococcal disease (IMD) caused by meningococcal bacteria (Neisseria meningitidis) strains A, C, W, and Y.
- FDA OKs Nucala for Hard-to-Treat COPD
May 23, 2025 — The FDA has approved the asthma drug Nucala for adults with chronic obstructive pulmonary disease (COPD).
- FDA Approves Novavax COVID Vaccine With Restricted Use
May 19, 2025 — The FDA has approved the Novavax COVID-19 vaccine, called Nuvaxovid, mainly for adults 65 or older, but people ages 12 to 64 with medical conditions that increase their risk of serious illness from COVID can also get the shot.