Drug News: 05 May 2025

May 5, 2025

Intriguing Science

Have you heard about jumping genes? These transposons make up 40% of the human genome and were once seen as harmless. Researchers are now studying how inhibiting their re-incorporation into chromosomes could control inflammation, neurodegenerative diseases, or aging. Development is underway for cancer and Alzheimer’s treatments, with promising phase 2 results.More information available in a recent article of Science.

European Medicine Agency (EMA)

The EMA’s Committee for Medicinal Products for Human Use (CHMP) held its monthly meeting from April 22 to 25, 2025. Here are the key takeaways:

Alyftrek (deutivacaftor / tezacaftor / vanzacaftor, Vertex Pharmaceuticals) received a positive opinion for marketing authorization for the treatment of cystic fibrosis in patients aged six years and older.

Attrogy (diflunisal, Purpose Pharma) received a positive opinion for the treatment of hereditary transthyretin-mediated amyloidosis.

Duvyzat (givinostat, ITF Therapeutics) received a positive recommendation for conditional marketing authorization as a treatment for Duchenne muscular dystrophy (DMD) ambulatory patients aged six and older.

Sephience (sepiapterin, PTC Therapeutics) received a positive opinion for the treatment of hyperphenylalaninaemia in adults and children with phenylketonuria.

Tepezza (teprotumumab, Amgen) received a positive opinion for the treatment of adults with moderate to severe Thyroid Eye Disease (TED).

Ziihera (zanidatamab, Jazz Pharmaceuticals) received a positive recommendation for conditional marketing authorization for the treatment of adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer.

Winlevi (clascoterone, Cassiopea) received a negative opinion for the treatment of acne vulgaris.

The CHMP adopted positive opinions for nine biosimilar medicines.CHMP meeting highlights.

US Food and Drug Administration (FDA)

Penpulimab-kcqx (Akeso BioPharma) was approved in combination with cisplatin or carboplatin and gemcitabine for the first-line treatment of adult patients with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). Akeso press release.

Zevaskyn (prademagene zamikeracel, Abeona Therapeutics) was approved for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Abeona Therapeutics press release.

Imaavy (nipocalimab-aahu, Johnson & Johnson) was approved for the treatment of generalized myasthenia gravis (gMG). J&J press release.

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 April 21 and May 4, 2025:

AstraZeneca announced the following:

Two Phase 3 studies (KALOS and LOGOS studies) of Breztri Aerosphere (budesonide/glycopyrronium/formoterol fumarate) in patients with uncontrolled asthma met all primary endpoints. AstraZeneca press release.
The discontinuation of the Phase III trial evaluating Truqap (capivasertib) in combination with docetaxel, and androgen-deprivation therapy (ADT)in patients with metastatic castration-resistant prostate cancer (mCRPC , following an interim analysis by the IDMC indicating the trial was unlikely to meet its dual primary endpoints. AstraZeneca press release.
Positive data from the Phase 3 trial of Enhertu (trastuzumab deruxtecan) in combination with pertuzumab for the treatment for patients with HER2-positive metastatic breast cancer. AstraZeneca press release.

Bristol Myers Squibb announced that the Phase 3 trial of Cobenfy (xanomeline and trospium chloride) as an adjunctive therapy in adults with inadequately controlled schizophrenia symptoms did not meet its primary endpoint. BMS press release.

Gilead Sciences reported positive results from the Phase 3 study of Trodelvy (sacituzumab govitecan-hziy) plus Keytruda (pembrolizumab, Merck) in patients with unresectable locally advanced or metastatic triple-negative breast cancer (mTNBC). Gilead press release.

Pfizer announced positive results from the Phase 3 trial of sasanlimab in patients with BCG-naïve, high-risk non-muscle invasive bladder cancer (NMIBC). Pfizer press release.

ALX Oncology announced that two Phase 2 trials of evorpacept plus Keytruda (pembrolizumab, Merck) with or without chemotherapy in in head and neck cancer did not meet primary endpoints. ALX Oncology press release.

Immunic announced positive data from its phase 2 trial of vidofludimus calcium (IMU-838) in patients with progressive multiple sclerosis (PMS). Immunic press release.

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