May 2024 Drug News, EMA, FDA approvals and new clinical data

June 4, 2024

European Medicine Agency (EMA)

The EMA’s Committee for Medicinal Products for Human Use (CHMP) had their monthly meeting the week from the 27th to 30th of May. Here are the key decisions:

Durveqtix (fidanacogene elaparvovec, Pfizer) received a positive CHMP opinion recommending approval for the gene therapy treatment for hemophilia B.
Adzynma (rADAMTS13, Takeda) was granted marketing authorisation under exceptional circumstances for the treatment of children and adult patients with congenital thrombotic thrombocytopenic purpura.
Akantior (polyhexanide, SIFI) received a positive opinion for the treatment of acanthamoeba keratitis.
Cejemly (sugemalimab, CStone) received a positive opinion for the treatment of adults with metastatic non-small-cell lung cancer.
Fluenz (influenza nasal vaccine live attenuated, AstraZeneca) received a positive opinion for the prophylaxis of influenza in children and adolescents aged 24 months to less than 18 years.
Ixchiq (chikungunya live vaccine live, Valneva), received a positive opinion to protect adults against disease caused by Chikungunya virus.
Zegalogue (dasiglucagon, Novo Nordisk) received a positive opinion for the treatment of severe hypoglycaemia in adults, adolescents, and children aged six years and over with diabetes mellitus.

The CHMP also recommended seven label extensions for already approved drugs: Dupixent, Eliquis, Kinpeygo, Livmarli, Skyrizi, Tagrisso, and Tevimbra. Additionally, CHMP recommended authorization for six generics or biosimilar including Avzivi (bevacizumab, Bio-Thera) a biosimilar to Roche’s Avastin. CHMP meeting highlights.

From safety perspective, The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) has called for the marketing suspension of drugs containing 17-hydroxyprogesterone caproate (17-OHPC) in Europe, citing concerns over both safety and efficacy. PRAC monthly meeting highlights.

US Food and Drug Administration (FDA)

In May, the FDA approved or granted designations to the following compounds:

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.
Tivdak (tisotumab vedotin-tftv, Pfizer) received full approval for the treatment of patients with recurrent or metastatic cervical cancer showing disease progression on or after chemotherapy. Pfizer press release.
Breyanzi (lisocabtagene maraleucel, BMS) was approved by the FDA for relapsed or refractory mantle cell lymphoma. FDA press release.
The FDA has approved mRESVIA (mRNA-1345, Moderna), an mRNA respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection. Moderna press release.
Imdelltra (tarlatamab-dlle, Amgen) has been approved by the FDA for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. Amgen press release.
The FDA has granted Breakthrough Therapy Designation for inavolisib in combination with palbociclib (Ibrance) and fulvestrant, for the treatment of adult patients with advanced hormone receptor-positive, HER2-negative breast cancer with a PIK3CA mutation. Roche press release.
The FDA granted accelerated approval to Retevmo (selpercatinib, Eli Lilly) for the treatment of paediatric patients two years and older with RET-altered metastatic thyroid cancer or solid tumors. FDA press release.

The FDA approved the following indication extensions, reformulation or biosimilars:

The FDA approved Onyda XR (clonidine hydrochloride, Tris Pharma) for treating ADHD as monotherapy or as adjunctive therapy to approved central nervous system stimulant medications in paediatric patients aged six years and older. Tris Pharma press release.
The FDA approved Yesafili (aflibercept-jbvf, Biocon Biologics) and Opuviz (aflibercept-yszy, Biogen) as the first interchangeable biosimilars to Eylea (aflibercept, Regeneron) for treating macular degeneration and other eye conditions (FDA press release). FDA press release.
Bkemv (eculizumab-aeeb, Amgen) received FDA approval as the first interchangeable biosimilar to Soliris (eculizumab, AstraZeneca) for treating patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).FDA press release.
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.
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.
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.
Austedo XR (deutetrabenazine, Teva) received FDA approval as a once-daily treatment for adult patients with tardive dyskinesia (TD) and Huntington’s disease (HD) chorea. Teva press release.
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.
The FDA extended the indication of Breyanzi (lisocabtagene maraleucel; liso-cel, BMS) to include the treatment of adult patients with relapsed or refractory follicular lymphoma. BMS press release.
The FDA approved a new subcutaneous route of administration of Benlysta (belimumab, GSK) for patients five years of age and older with active systemic lupus erythematosus (SLE). GSK press release.

The following NDA/BLA has been rejected by the FDA:

Elevar Therapeutics (subsidiary of HLB) and Jiangsu Hengrui Pharmaceuticals announced that the FDA rejected their drug combination (rivoceranib/ camrelizumab) for the treatment of liver cancer. The Complete Response Letter (CRL) cited issues chemistry, manufacturing, and controls (CMC) and logistic issues to audit some of the key clinical sites.
The FDA rejected Dynavax’s application for Heplisav-B (Hepatitis B vaccine) to be used in adults undergoing hemodialysis. The CRL stated that the application did not provide sufficient data to support a full evaluation of effectiveness or safety. Dynavax press release.

The following NDA/BLA review was delayed:

Ascendis Therapeutics announced the FDA postponed their decision on TransCon PTH (palopegteriparatide) for the treatment adults with hypoparathyroidism. Ascendis press release.

Clinical trials newly reported data

In neurology trials, here are the data reported in May 2024:

Biogen and Ionis have decided to terminate development of BIIB105 (ION541) for the treatment of amyotrophic lateral sclerosis (ALS) based on results from its Phase 1/2 trial. Biogen press release.
Larimar Therapeutics announced that the FDA has removed the Partial Clinical Hold for nomlabofusp (CTI-1601) for the treatment of Friedreich’s Ataxia. Larimar will continue the open-label extension study with results expected in Q4 this year. Larimar press release.
NS Pharma has announced preliminary results from the global Phase 3 study of viltolarsen (NS-065 / NCNP-01) for Duchenne muscular dystrophy (DMD) treatment. The study reported a numerical improvement. NS Pharma press release (pdf).
Neuren Pharmaceuticals announced positive data from its Phase 2 trial of NNZ-2591 for the treatment of children with Pitt Hopkins syndrome (PTHS). Neuren press release (pdf).
Otsuka Pharmaceutical and Lundbeck Pharmaceuticals announced results from three clinical trials evaluating the combination of brexpiprazole and sertraline in treating adults with post-traumatic stress disorder (PTSD). Otsuka press release (pdf).
Johnson & Johnson (J&J) has announced that seltorexant met all the primary and secondary endpoints in a Phase 3 trial in major depressive disorder (MDD). J&J press release.
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.
Evenamide (Newron Pharmaceuticals) , an add-on schizophrenia treatment, , has met both primary and secondary endpoints in a Phase 2/3 trial. Newron press release.

The following oncology/immunology trials reported data in May 2024:

Merck announced the discontinuation of a Phase 3 study investigating the co-formulation of vibostolimab and pembrolizumab (Keytruda) in patients with resected high-risk melanoma, due to immune-mediated adverse events. Merck press release.
BMS announced new four-year results from the long-term extension (LTE) trial of Sotyktu (deucravacitinib) in adult patients with moderate-to-severe plaque psoriasis. BMS press release.
AstraZeneca has announced that the Phase 3 trial of sipavibart (AZD3152) met the primary endpoints in preventing Covid-19 among immunocompromised individuals. AstraZeneca press release.
Faron Pharmaceuticals announced initial positive data from the Phase 2 trial of bexmarilimab in combination with azacytidine in relapsed/refractory myelodysplastic syndrome (r/r MDS). Faron press release.
J&J reported positive news from two Phase 3 studies of Tremfya (guselkumab). Tremfya has met its primary endpoint and all major secondary endpoints as a maintenance therapy in patients with moderate to severe active ulcerative colitis. Tremfya was also superior to both Stelara (ustekinumab) and placebo in all endoscopic endpoints in adult patients with moderately to severely active Crohn’s disease. J&J press releases [1], [2].
Nimbus Therapeutics released positive results from the ongoing Phase 1/2 trial of NDI-101150 for treating adults with advanced solid tumours. Nimbus press release.
Human Immunology Biosciences (HI-Bio) reported positive outcomes from the Phase 2 trial of felzartamab for the treatment of late antibody-mediated rejection (AMR) in kidney transplant recipients. Hi-Bio press release.
Innovent Biologics reported that its Phase 3 study of picankibart (IBI112) for treating moderate to severe plaque psoriasis met its primary and all key secondary endpoints. Innovent press release.
Eli Lilly shared positive data from the Phase 3 study of mirikizumab for treating moderately to severely active Crohn’s disease. Eli Lilly’s press release.
AstraZeneca and Daiichi Sankyo shared positive results from the Phase 3 trial of datopotamab deruxtecan in patients with advanced nonsquamous non-small cell lung cancer (NSCLC). AstraZeneca press release.
Gilead Sciences announced that Trodelvy (sacituzumab govitecan-hziy; SG) failed to meet the primary endpoint in its Phase 3 study in locally advanced or metastatic urothelial cancer (mUC). Gilead press release.
Novartis presented positive results from the Phase 3 study of Scemblix (asciminib) in patients with newly diagnosed chronic myeloid leukemia (CML). Novartis press release.
Summit Therapeutics reported that its Phase 3 trial of ivonescimab in patients with PD-L1 positive non-small cell lung cancer (NSCLC) in China met the primary endpoint. Summit press release (pdf).
Enhertu (trastuzumab deruxtecan, AstraZeneca) showed positive data in a phase 3 in patients with HR-positive, HER2-low metastatic breast cancer. AstraZeneca press release.
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.
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.
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.
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.
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.

The following cadio-metabolic trials reported data in May 2024:

Eli Lilly shared positive results from two Phase 3 clinical trials evaluating once-weekly insulin efsitora alfa (efsitora) in adults with type 2 diabetes, including those who are insulin-naive and those who require multiple daily insulin injections. Eli Lilly press release.
Cardurion Pharmaceuticals reported that CRD-740 has met the primary endpoint of its Phase 2a study in heart failure patients. Cardurion press release.
Cytokinetics shared positive results from the Phase 3 trial of aficamten in patients with obstructive hypertrophic cardiomyopathy (HCM). The company plans to seek regulatory approval in the US and Europe later this year. Cytokinetics press release.
Tango Therapeutics has terminated the development of its TNG348 programme after data emerged from the Phase 1/2 trial indicated that the therapy may cause liver dysfunction. Tango press release.

In other indications, the following data were reported in May 2024:

Insmed announced that its Phase 3 study of brensocatib for treating patients with non-cystic fibrosis bronchiectasis met all its primary and key secondary endpoints. Insmed press release.
Bayer announced that their non-hormonal therapy, elinzanetant, met primary and secondary endpoints in two Phase 3 trials by reducing the frequency and severity of hot flashes in in post-menopausal women. Bayer press release.
Novo Nordisk shared positive results from a Phase 3a trial of subcutaneous Mim8 in treating haemophilia A. Novo Nordisk press release.
Vanda Pharmaceuticals reported positive outcomes from the Phase 3 study of tradipitant for the treatment of motion sickness. Vanda press release (pdf).
GSK announced positive data from two Phase 3 trials of depemokimab for the treatment of asthma. Depemokimab met primary endpoints in both trials. GSK press release.
Sanofi shared positive results from a Phase 2 trial of rilzabrutinib for the treatment of adults with uncontrolled moderate-to-severe asthma. Sanofi press release.
Novartis released new data from two Phase 3 trials confirming the sustained efficacy and long-term safety of remibrutinib in treating chronic spontaneous urticaria (CSU). Novartis press release.
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.