Drug News: 14 July 2025

Intriguing science

Deep Brain Stimulation is an intervention that is used in s Parkinson’s Disease, Tourette, epilepsy, refractory pain and other CNS conditions. Since 3 decades, the devices needed to be programmed to deliver specific stimuli via electrodes, and the clinician could adapt the delivered signal depending on the patient presentation.

After several years of development, a new function is now being approved: adaptative DBS (aDBS). This new feature will modulate the signal based on the actual signals read through implemented probes, and the promise is to better control the symptoms of interest.  The regulators clearance will be for patients with Parkinson’s disease.

For more information, read this article from Nature: doi: https://doi.org/10.1038/d41586-025-02224-3

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European Medicine Agency (EMA)

The EMA’s Committee for Pharmacovigilance Risk Assessment Committee (PRAC) held its monthly meeting from July 7 to 10, 2025. Here are the key takeaways:

1. Ixchiq (Live Attenuated Chikungunya Vaccine), safety review completed: The temporary restriction on vaccinating individuals aged 65 and above has been lifted.
2. Updated information regarding the risk of encephalitis with Varicella Vaccines: Varilrix and Varivax.
3. Clozapine: Revised Recommendations for Blood Count Monitoring
4. Valproate and Potential Risk of Neurodevelopmental Disorders

Further data and analysis have been requested from the marketing authorisation holders to better understand discrepancies across studies. PRAC meeting highlights.

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US Food and Drug Administration (FDA)

1. Gamifant (emapalumab-lzsg, Sobi) was approved for the treatment of adult and pediatric patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still’s disease. Sobi press release.
2. Gammagard Liquid ERC (immune globulin infusion (human), Takeda) was approved as replacement therapy for patients two years of age and older with primary immunodeficiency (PI). Takeda press release.
3. Spikevax (COVID-19 vaccine, Moderna) received full approval in children 6 months through 11 years of age who are at increased risk for COVID-19 disease. Moderna press release.
4. Tyzavan (Vancomycin Injection, USP, Hikma Pharmaceuticals) was approved for the treatment of septicemia, infective endocarditis, skin and skin structure infections, bone infections, and lower respiratory tract infections in adults and children 1 month or older. Hikma press release.
5. Kerendia (finerenone, Bayer) was approved for the treatment of adult patients with heart failure (HF) and a left ventricular ejection fraction (LVEF) of ≥40%. Bayer press release.
6. Lynozyfic (linvoseltamab-gcpt, Regeneron) received FDA accelerated approval to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM). Regeneron press release.
7. Zegfrovy (sunvozertinib, Dizal (Jiangsu) Pharmaceutical) received FDA accelerated approval for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. FDA press release.
8. Ekterly (sebetralstat, KalVista Pharma) was approved for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older. KalVista press release.
9. Kisunla (donanemab-azbt, Eli Lilly) received a label update for the treatment of adults with early symptomatic Alzheimer’s disease (AD). Lilly press release.
10. UX111 (ABO-102, Ultragenyx Pharmaceutical) was rejected as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) issues. Ultragenyx press release.
11. Oxylanthanum Carbonate (OLC, Unicycive Therapeutics) was rejected for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. The CRL cited deficiencies previously identified at a third-party manufacturing vendor. Unicycive Therapeutics press release.

Deramiocel (CAP-1002, Capricor Therapeutics) was rejected for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The CRL cited agency concerns with clinical data and certain manufacturing commitments. Capricor press release.

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New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between June 30 and July 14, 2025:

1. Organon announced that the Phase 2 study evaluating the OG-6219 in endometriosis-related pain did not meet its primary endpoint. Organon press release.
2. Cogent Biosciences shared positive results from the Phase 2 trial of bezuclastinib in patients with non-advanced systemic mastocytosis (NonAdvSM). Cogent Biosciences press release.
3. Apogee Therapeutics reported positive data from the Phase 2 trial of APG777 in patients with moderate-to-severe atopic dermatitis (AD). Apogee press release.
4. Takeda announced that two Phase 3 studies of oveporexton (TAK-861) in narcolepsy type 1 (NT1) met all primary and secondary endpoints. Takeda press release.
5. AstraZeneca announced that the Phase III trial of baxdrostat in patients with uncontrolled or treatment resistant hypertension met the primary and all secondary endpoints. AstraZeneca press release.
6. Amgen reported that the Phase 3 trial evaluating bemarituzumab plus chemotherapy (mFOLFOX6) in patients with unresectable locally advanced or metastatic gastric or gastroesophageal junction (G/GEJ) cancer with FGFR2b overexpression met its primary endpoint. Amgen press release.
7. Pfizer and Astellas Pharma shared positive results from the Phase 3 study evaluating Xtandi (enzalutamide), in combination with leuprolide and as a monotherapy, in men with non-metastatic hormone-sensitive prostate cancer. Pfizer press release.
8. Rhythm Pharmaceuticals reported positive data from its Phase 2 trial of bivamelagon (formerly LB54640) in patients with acquired hypothalamic obesity. Rhythm press release.
9. LEO Pharma announced positive results from the Phase 3b trial of tralokinumab for the treatment of adults with moderate-to-severe atopic dermatitis on the hands. LEO Pharma press release.
10. Taiho Pharmaceutical announced that the Phase 3 study of TAS-205 in patients with Duchenne muscular dystrophy (DMD) did not meet its primary endpoint. Taiho press release.