FDA Weekly Review: Bayer, Daiichi Sankyo, AstraZeneca, Gamida Cell and More

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The U.S. Food and Drug Administration has had a busy week, accepting drug applications, approving clinical trials and granting various special designations. Continue reading for more details.

Bayer received a nod from the FDA for the use of Nubeqa (darolutamide) in combination with docetaxel chemotherapy for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC). This follows a previous approval of Nubeqa in non-metastatic castration-resistant prostate cancer (nmCRPC). The newest approval is based on the Phase III ARASENS trial, which studied Nubeqa plus androgen deprivation therapy (ADT) and docetaxel compared to ADT and docetaxel.

The FDA approved Daiichi Sankyo and AstraZeneca’s ENHERTU for another indication. It is now approved for the treatment of adult patients with a range of HER2 expressions, including those with unresectable or metastatic HER2 low breast cancer. This additional indication was based on the DESTINY-Breast04 Phase III trial. 

Gamida Cell Ltdannounced that the FDA accepted its Biologics License Application for omidubicel. The therapy is being developed for the treatment of patients with blood cancers who need an allogeneic hematopoietic stem cell transplant.

Cellectis reported the FDA cleared its Investigational New Drug (IND) application to initiate a Phase I/IIa trial of UCART20x22 for patients with relapsed or refractory Non-Hodgkin Lymphoma (r/r NHL). UCART20x22 features TALEN-mediated disruptions of the TRAC gene and of the CD52 gene.

Scynexis announced that the FDA accepted its sNDA for Brexafemme for the treatment of vulvovaginal candidiasis (WC) and the prevention of recurrent WC. It was accepted for Priority Review with a PDUFA date of November 30, 2022.

The Janssen Pharmaceutical Companies of Johnson & Johnson announced the FDA approved Stelara (ustekinumab) for the treatment of children six and older with active psoriatic arthritis (PsA). The drug is a fully human monoclonal antibody that selectively inhibits IL-12 and IL-23.

Krystal Biotech reported that the FDA had accepted its IND application for KB407 for cystic fibrosis. KB407 is a modified HSV-1 vector carrying two copies of the CF transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.

Beam Therapeutics announced the FDA placed a clinical hold on its IND application for BEAM-201 for relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL). No reason for the hold was supplied. BEAM-201 is a donor-derived CAR-T cell therapy that has undergone multiplex base editing to become universally compatible and resistant to rejection by the host.

Celyad Oncology reported the FDA has lifted its clinical hold on the Phase Ib KEYNOTE-B79 trial. The hold was placed in February after two patient deaths. CYAD-101 is an allogeneic CAR-T cell therapy designed to express the NKG2D receptor, generally found on NK cells, and the novel inhibitory peptide TIM. It is being tested with Merck’s Keytruda.

Marius Pharmaceuticals announced that the FDA approved Kyzatrex (testosterone undecanoate) as an oral testosterone replacement therapy for adult males for conditions associated with deficiency or absence of endogenous testosterone (hypogonadism).

MAIA Biotechnology received Orphan Drug Designation from the FDA for THIO. THIO is a telomere-targeting agent in development for multiple cancers, including small-cell lung cancer.

Biosight was granted Orphan Drug Designation by the FDA for asparcytarabine for treatment of myelodysplastic syndromes. Asparcytarabine is a novel proprietary anti-metabolite made up of cytarabine covalently bound to asparagine.

Calidi Biotherapeutics announced that City of Hope received FDA authorization to proceed with a Phase I trial of Calidi’s oncolytic virotherapy platform, NSC-CRAd-S-pk7. The therapy is made up of tumor-tropic neural stem cells delivering an oncolytic adenovirus selectively to tumor sites in patients with recurrent high-grade glioma.

Twist Bioscience and Biotia’s EUA for its Next-Generation Sequencing Assay for SARS-COV-2 received expanded authorization.

Avenge Bio announced the FDA cleared its IND for AVB-001 in peritoneal malignancies. The drug is designed to secrete native IL-2 in immune-activating alginate capsules.

Amylyx Pharmaceuticals reported that the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee will reconvene to review its NDA for AMX0035 (sodium phenylbutyrate and taurursodiol) for ALS. It previously met on March 30 but is reconvening to discuss additional data analysis.

Allergan Aesthetics, an AbbVie company, announced the FDA approved Juvederm Volux XC for the improvement of jawline definition in adults over the age of 21 with moderate to severe loss of jawline definition.

AnHeart Therapeutics received Breakthrough Therapy Designation from the FDA for taletrectinib for adults with advanced or metastatic ROS-1-positive non-small cell lung cancer (NSCLC) who are ROS1 TKI treatment naïve or previously treated with crizotinib. Taletrectinib is a ROS-1 inhibitor.

OliX Pharmaceuticals’s IND for OLX10212 was approved for age-related macular degeneration. OLX10212 targets inflammation pathways that play a key role in geographic atrophy and neovascular AMD. It is for a Phase I trial.

Sumitomo Pharma Oncology reported the FDA granted Orphan Drug Designation for DSP-5336 for acute myeloid leukemia. The drug is a small molecule inhibitor against the binding of minin and mixed-lineage leukemia (MLL) protein.

Pulse Biosciences announced they had received FDA clearance of expanded energy settings for use with the family of CellFX System treatments tips in dermatology. CellFX System uses the company’s Nano-Pulse Stimulation technology to deliver electrical energy to clear cells.

Vaxcyte reported the FDA granted Fast Track designation to VAX-24, its 24-valent pneumococcal conjugate vaccine candidate for adults 18 and older.

GenBody America announced that the FDA granted an extension of the shelf life for its COVID-19 Ag (antigen) test from 12 months to 21 months when stored at 2 to 30 degrees C (35-86 degrees F).

Acadia Pharmaceuticals announced they had received a Complete Response Letter from the FDA for its sNDA for Nuplazid (pimavanserin) for the treatment of hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). The agency recommended that the company run another trial in ADP.

ProfoundBio received the go-ahead from the FDA for its IND to evaluate PRO1184 in patients with advanced cancer in a Phase I trial. The drug is an antibody-drug conjugate made up of a folate receptor alpha directed antibody conjugated to an exatecan payload with a proprietary hydrophilic linker. 

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