A Roundup of Global Drug Approvals in December 2023

To combat the rising burden of chronic diseases, the pharmaceutical industry has been making continuous and dedicated efforts to bring accurate, effective, and personalized solutions to patients. While researchers play a pivotal role in developing drugs, the regulatory authorities are equally critical to ensure the safety and quality of drugs being brought to the consumers. Signing 2023 off on a momentous note, the pharmaceutical industry observed the approval of several new innovative drugs.

Here are some of the key drug approvals across the world in December 2023.

Filsuvez Topical Receives FDA Nod for Epidermolysis Bullosa Treatment

Filsuvez (birch triterpenes), developed by Chiesi Global Rare Disease is a topical gel for the treatment of epidermolysis bullosa. The topical gel, made from the dry extract of two species of birch bark can also be applied to wounds during home dressing. The approval was based on results from the Phase 3 EASE trial, which included 223 patients across 3 countries. Approximately 41.3% patients out of all those who were treated with the medication showed complete target wound closure within 45 days.

Epidermolysis bullosa, a rare disease, has an incidence rate of approximately 45 per million live births, out of which dystrophic bullosa has a prevalence rate of 15.58 cases per million live births. The approval indicates that the medication can be used for patients aged 6 months and above for both junctional and dystrophic epidermolysis bullosa.

Japan Approves New Drug for Treating Alzheimer’s Disease

LEQEMBI® Intravenous Infusion (200 mg, 500mg, lecanemab), a humanized anti- soluble aggregated amyloid-beta (Aβ) monoclonal antibody received an approval to be sold in Japan in December 2023. The drug is expected to be co-promoted by Eisai Co., Ltd., and Biogen Inc., with Eisai as the Marketing Authorisation Holder.

LEQEMBI is the first and only approved treatment alternative that has reduced disease progression. It selectively binds to soluble (protofibrils) amyloid-beta aggregates as well as insoluble (fibrils) amyloid-beta aggregates and reduces Aβ protofibrils and Aβ plaques in the brain. With this launch, Japan becomes the second country to have LEQEMBI in the markets, only after the United States.

Merck Develops Novel Treatment Drug for Renal Cell Carcinoma

The FDA approved WELIREG® (belzutifan) for patients affected by renal cell carcinoma. Welireg, an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor was authorised on the basis of clinically significant results from LITESPARK-005 clinical trial, that included 746 patients. In the trial, WELIREG demonstrated superior progression-free survival against everolimus in advanced renal cell carcinoma. The treatment was followed by both a PD-1 or PD-L1 checkpoint inhibitor and a VEGF receptor targeted therapy, administered in combination or in sequence.

Renal cell carcinoma is the most prevalent type of kidney cancer, which is twice as common in men as compared to women. Around 15% of the patients are diagnosed at an advanced stage, making the treatment critical and difficult several folds.

EMA Signals Green Light for Duchenne Muscular Dystrophy Novel Drug

On December 18th, 2023, Santhera Pharmaceuticals revealed that AGAMREE® (vamorolone), their latest drug for Duchenne muscular dystrophy received European Union approval. The authorisation was based on the positive data from the VISION-DMD study and open label studies wherein vamorolone was administered at doses between 2 and 6 mg/kg/day for an overall treatment period of around 30 months. The medication is expected to reach the German markets by Q1 2024.

The action mechanism of the drug is based on binding to the same receptor as glucocorticoids and modifying its downstream activity. As a result, it has been posed as an alternative to existing corticosteroids-based treatment. In addition, the drug doesn’t have any negative effects on the bone metabolism like other drugs.

China Authorises Sugemalimab (Cejemly®) for Treating Esophageal Squamous Cell Carcinoma

The National Medical Products Administration (NMPA) of China approved supplemental biologics license application (sBLA) for CStone’s Sugemalimab (Cejemly®), an anti-PD-L1 monoclonal antibody to treat locally advanced, recurrent, or metastatic esophageal squamous cell carcinoma. The drug when used in combination with fluorouracil and platinum-based chemotherapy showed overall survival and progression-free survival in the GEMSTONE-304 study.

Esophageal squamous cell carcinoma has a high prevalence rate in China, with around 70% of the patients progressing towards locally advanced or advanced stages at the time of initial diagnosis. In addition, the drug is also used for treatment of unresectable stage 3 non-small cell lung cancer.

Indolent Systemic Mastocytosis Treatment Receives EMA’s Nod

The European Commission approved Blueprint Medicines Corporation’s AYVAKYT® (avapritinib) for the treatment of indolent systemic mastocytosis (ISM) in adults. It is the first and only approved therapy for ISM patients in Europe and helps in treating moderate to severe symptoms which may significantly impair the quality of life. The approval follows the positive opinion of CHMP (Committee for Medicinal Products for Human Use), and statistically positive data and durable clinical benefits shown in the PIONEER trial.

Systemic mastocytosis is a rare hematologic disorder that affects approximately 40,000 people in the European region. It works by selectively targeting KIT D816V, a primary driver of the disease.

AstraZeneca’s WAINUA (eplontersen) for ATTRv-PN Receives FDA Regulatory Approval

In December 2023, Ionis Pharmaceuticals, Inc., and AstraZeneca's WAINUA™ (eplontersen) received FDA approval for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis (commonly referred as hATTR-PN or ATTRv-PN) in adults. WAINUA is a ligand-conjugated antisense (LICA) medicine that works by reducing the production of TTR protein at its source. It is the only approved medicine that can be self-administered via an auto-injector.

Patients affected by ATTRv-PN experience loss of motor skills, owing to debilitating nerve damage throughout their body. The damage may lead to a disability within five years of diagnosis. The approval is based on positive 35-week interim analysis from the Phase 3 NEURO-TTRansform study and will be made available in the United States market by January 2024.

FDA Approves Novartis’ Fabhalta® (iptacopan) for Paroxysmal Nocturnal Hemoglobinuria

Fabhalta® (iptacopan) received FDA approval for treating paroxysmal nocturnal hemoglobinuria (PNH). It is the first oral monotherapy and a Factor B inhibitor that prevents red blood cell destruction within as well as outside the blood vessels. Intravascular haemolysis (destruction of RBCs withing the blood vessels) and extravascular haemolysis (destruction of RBCs mostly in the spleen and liver) are common indications of PNH.

As per the results of the Phase III APPLY-PNH clinical trial in patients with residual anaemia, the drug also improved haemoglobin levels in the body. PNH affects approximately 10-20 people per million worldwide. The disease is diagnosed in people aged around 30-40 years of age, however, it can develop at any age.

As research and development in pharmaceutical organizations continue to evolve, the impact on patient care is set to witness magnanimous changes in the forthcoming years.