European Medicine Agency (EMA)
No new decision this week.
Of note, Apellis Pharmaceuticals, after winning a case in the European Court, saw their MAA for intravitreal pegcetacoplan in the treatment pf geographic atrophy reset to the final stage of the assessment (day 180). Apellis press release.
US Food and Drug Administration (FDA)
The FDA approved the following compounds:
1. Xolremdi (mavorixafor, X4 Pharmaceuticals) in patients of 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) and a Rare Pediatric Disease Priority Review Voucher (PRV) with the approval. This is the first treatment of this indication. X4 press release.
2. Tivdak (tisotumab vedotin-tftv, Pfizer) received full approval from the FDA for the treatment of patients with recurrent or metastatic cervical cancer showing disease progression on or after chemotherapy. Pfizer press release.
The FDA also issued the following decisions:
1. Approval of Hercessi (trastuzumab-strf, Accord BioPharma), a biosimilar to Roche’s Herceptin to treat HER2-overexposing breast and gastric or gastroesophageal junction adenocarcinoma. Accord press release.
2. Approval of the updated label for Lupkynis (voclosporin, Aurinia Pharmaceuticals) to include three-year safety and efficacy data from an extension study. Aurinia press release.
3. Libervant (Diazepam, Aquestive Therapeutics) buccal film was approved to treat seizure clusters and acute repetitive seizures in patients ages 2 to 5 with epilepsy. This makes it the only orally administered rescue product to treat these conditions. Aquestive press release.
4. Ingrezza (valbenazine, Neurocrine Biosciences) sprinkle capsules was approved for the treatment of adults with tardive dyskinesia and chorea associated with Huntington’s disease. Neurocrine 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 the 29th of April and 5th of May 2024:
1. Enhertu (trastuzumab deruxtecan, AstraZeneca) showed positive data in a phase 3 in patients with HR-positive, HER2-low metastatic breast cancer. AstraZeneca press release.
2. ADX71149 (JNJ-40411813, Addex Therapeutics) failed to meet primary endpoints in a Phase 2 trial in patients with focal onset seizures with suboptimal response to levetiracetam or brivaracetam. Addex did not provide additional information regarding the drug’s future development. Addex press release.
3. A combination of Ampligen (rintatolimod, AIM ImmunoTech) and Imfinzi (durvalumab, AstraZeneca) showed positive data in the treatment of late-stage pancreatic cancer patients. AIM ImmunoTech press release.
4. The 21-valent V116 vaccine (Merck) showed positive data in a phase 2 trial designed to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults who have not previously received a pneumococcal vaccine. Merck press release.
5. Evenamide (Newron Pharmaceuticals) , an add-on schizophrenia treatment, , has met both primary and secondary endpoints in a Phase 2/3 trial. Newron press release.
6. Another positive data for Keytruda (Merck) in a Phase 3 trial of (pembrolizumab) plus trastuzumab and chemotherapy for the treatment of patients with HER2-positive advanced gastric or gastroesophageal junction adenocarcinoma. Merck press release.
7. Positive results from a Phase 1/2 study evaluating its measles and rubella microneedle patch (Micron Biomedical). The needle-free vaccine demonstrated safety and immunogenicity in children. Micron press release.
8. Atsena Therapeutics announced positive preliminary data from a Phase 1/2 trial of ATSN-201 gene therapy for the treatment of X-linked Retinoschisis (XLRS). Atsena press release.
9. Positive interim results from a Phase 3 trial of Calquence (acalabrutinib, AstraZeneca) in combination with standard chemoimmunotherapy for treating mantle cell lymphoma (MCL). AstraZeneca press release.
