An Overview of Latest Global Drug Approvals in January 2024

With the rising prevalence of chronic diseases, the global biopharmaceuticals market is also witnessing a significant upswing. According to EMR calculations, the biopharmaceuticals market size is anticipated to reach a value of USD 845.5 billion by 2032 with a steady CAGR of 8.2%. Each drug undergoes several stringent trials until it finally reaches the market to be used by the patient. In the process, the regulatory bodies play a critical role in maintaining the efficacy and potency of drugs.

Here are the Latest Global Drug Approvals in January 2024, Poised to Transform the Medical Landscape Ahead.

FDA Offers Green Signal for New Metastatic Urothelial Carcinoma Treatment

The U.S. FDA approved the application of Janssen Biotech’s Balversa (erdafitinib) for treating locally advanced or metastatic bladder cancer (urothelial carcinoma) in patients on January 19th, 2024. The drug is particularly applicable on patients who cannot get their tumor removed via surgery, have a certain category of abnormal FGFR gene and have tried minimum one other medicine (which did not show any significant results).

The approval was based on the positive results from the BLC3001 Cohort 1 Study. It was a randomized, open-label trial of 266 patients with selected FGFR3 alterations. The set of patients had also received prior systemic treatments, including a PD-1 or PD-L1 inhibitor. As per the results, the confirmed ORR (overall response rate) was 35.3% in patients who received the treatment. It can be administered orally (in 8mg concentration), and the dosage can be increased up to 9 mg once daily, based on the tolerability in the patients.

New Hope for Treating Urinary Tract Infection as EMA Authorizes Exblifep

On 26th January 2024, the European Medicines Agency (EMA) announced that Exblifep (cefepime-enmetazobactam) can be marketed across the Europe for treating adults suffering from urinary tract infections (UTIs) and hospital acquired pneumonia. Urinary tract infections are amongst the most common infections in women.It is reported that 50% to 60% of the entire female population experience the disease once in their lifetime. A combination of cephalosporin, and enmetazobactam, Exblifep is an extended-spectrum beta-lactamase inhibitor.

The authorisation is based on the results from phase 3 ALLIUM clinical trial, which demonstrated the overall effectiveness of Exblifep (79.1%) over piperacillin-tazobactam (58.9%), marking the treatment difference to be 21.2%. The drug is available as a 2g/0.5g powder for administration.

Japan Allows Novel Paroxysmal Nocturnal Haemoglobinuria (PNH) Treatment

On 19th January 2024, the Japanese Ministry of Health, Labour, and Welfare (MHLW) approved Voydeya (danicopan) in combination with C5 inhibitor therapy for treating paroxysmal nocturnal haemoglobinuria (PNH). Voydeya is an oral Factor D inhibitor, which has been manufactured as an add-on to Ultomiris or Soliris (proven standard of care for PNH treatment). The results were based on the positive trials from ALPHA Phase III trial, wherein the initial oral dose of danicopan was 150 mg in patients. Eventually, the dosage was increased to 200 mg thrice a day, based on positive clinical responses. It was well tolerated in patients and showed no new safety concerns in the patients.

PNH is a severe and rare blood disorder, leading to the destruction of red blood cells, ultimately resulting in organ damage and premature death.

Ryzneuta Receives Marketing Authorisation for Neutropenia Treatment

The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion to market Ryzneuta for treating neutropenia, and chemotherapy associated febrile neutropenia on 25th January 2024. As per EMR calculations, the global neutropenia treatment market is poised to grow at a CAGR of 5.3% and attain a value of approximately USD 25.09 billion by 2032.

With efbemalenograstim alfa as an active substance, the drug boosts the production and differentiation of mature and functionally active neutrophils from bone marrow precursor cells to combat the declining number of neutrophils in the bloodstream. Consequently, it leads to the duration reduction of neutropenia. It will be made available in 20 mg solution with injections as the route of administration.

FDA Approves Novel Medication for Molluscum Contagiosum Treatment

On January 5th, 2024, Ligand Pharmaceutical’s ZELSUVMI ™ (berdazimer topical gel, 10.3%) gained the United States FDA approval for treating molluscum contagiosum (molluscum) in adult as well as pediatric patients (aged one year or older). The treatment is marked as the first FDA approved treatment for the highly contagious and infectious diseases, affecting around 6 million Americans (primarily children) every year.

Infused with nitric oxide (which has been shown to offer antiviral properties), ZELSUVMI demonstrated significant efficacy in two Phase 3 trials, namely, B-SIMPLE 4, and B-SIMPLE 2. The B-SIMPLE program entailed 1598 patients. In the trials, it reduced lesion counts and was well tolerated on patients.

Niapelf for Schizophrenia Treatment Receives EMA’s Marketing Approval

On January 25th, 2024, the Committee for Medicinal Products for Human Use (CHMP) recommended the marketing authorisation of Niapelf, intended for schizophrenia treatment. It is a generic of Xeplion and has indicated satisfactory quality and bioequivalence to the reference product (Xeplion). Paliperidone is an active substance present in the drug and acts as a primary active metabolite of risperidone (commonly used in schizophrenia treatment). The therapeutic effect is mediated through antagonist activity at D2- and 5-HT2A receptors.

Currently, around 24 million are affected by schizophrenia, with 1 out of 300 individuals residing in every country. As a result, developing effective schizophrenia treatments is a major area of attention amongst scientists. According to EMR estimations, global schizophrenia drugs market size is anticipated to rise significantly in the forecast period with a CAGR of 5.4% and reach a value of USD 12.97 billion by 2032. The recent approval of Neuraxpharm Pharmaceuticals’ Niapelf is expected to offer lucrative solutions to those affected by the disease.

China Approves Beyfortus for Treating RSV Disease in Infants

Beyfortus (nirsevimab), a monoclonal antibody developed by AstraZeneca and Sanofi received National Medical Products Administration (NMPA) approval for preventing respiratory syncytial virus (RSV)  lower respiratory tract infection (LRTI) in infants on 2nd January 2024. It is the first approved preventive alternative for infants and a leading cause of hospitalisation as well.

If left undiagnosed, the RSV virus can cause lower respiratory tract disease (LRTD), along with bronchitis and pneumonia. The authorisation is based on Phase IIb (Trial 03) study results, which is a randomised, placebo-controlled trial. Owing to the efficacy and urgent need to mitigate RSV associated morbidities, the drug has been granted regulatory designations to allow accelerated development by various key regulatory agencies across the globe, including Breakthrough Therapy Designation and Priority Review Designation by the China Center for Drug Evaluation under the National Medical Products Administration.

As the healthcare and pharma industry continues to progress with vital technological advancements, the market is expected to witness expediated development and approvals of multiple drugs. The key area of focus is on rare and fatal diseases, so as to provide effective solutions and address unmet needs of millions of patients across the globe.