Vaccine Sector: New Progress and Market Expansion

In the vaccine sector, Novavax's LP.8.1-formulated COVID-19 vaccine, Nuvaxovid, has received regulatory approval in Japan. It is indicated for primary immunization (first and second doses) in individuals aged 6 years and above, as well as booster doses in those aged 12 years and above. Pursuant to the 2020 license agreement with Takeda, this approval has triggered an undisclosed milestone payment from Takeda to Novavax, while Takeda is responsible for the manufacturing and commercialization of the vaccine in Japan.

Moderna's updated Spikevax COVID-19 vaccine has also made new headway: Canada approved its version targeting the currently prevalent SARS-CoV-2 LP.8.1 variant. Produced at Moderna's active pharmaceutical ingredient (API) facility in Quebec and filled and finished by Novocol Pharma in Ontario, this vaccine is suitable for individuals aged 6 months and above. Additionally, the updated vaccine obtained approval in Europe four weeks prior, and in May, the U.S. FDA approved its next-generation COVID-19 vaccine, mNEXSPIKE, for adults aged 65 years and above, as well as for individuals aged 12 to 64 years with serious COVID-19 risk factors.

Valneva's chikungunya vaccine, Ixchiq, has secured a broader use authorization in Canada, expanded to include individuals aged 12 years and above. Newly added antibody persistence data shows that 97% of patients maintain an immune response for 24 months, and among adolescents, a single dose of the vaccine elicits a high and durable immune response in 99.1% of recipients at 6 months. However, the European Medicines Agency (EMA) had previously imposed a temporary restriction on Ixchiq's use in individuals aged 65 years and above due to serious adverse events reported in this population. Although the restriction has now been lifted, the EMA recommends its use only when the infection risk is significant and after weighing the benefits and risks.

Cancer Treatment: Multiple Drug Approvals and Indication Expansions

The cancer treatment sector has seen a string of positive developments. AstraZeneca and Daiichi Sankyo's Datroway obtained approval in China for the treatment of adult patients with unresectable or metastatic HR-positive, HER2-negative breast cancer who have previously received endocrine therapy and at least one line of advanced chemotherapy. This approval is based on data from the phase III TROPION-Breast01 study, which showed that Datroway reduced the risk of disease progression or death by 37% compared to the investigator's chosen chemotherapy regimen.

Astellas' Padcev in combination with Merck & Co.'s Keytruda has received support from the UK's National Institute for Health and Care Excellence (NICE) for use in patients with unresectable or metastatic urothelial carcinoma who are eligible for platinum-containing chemotherapy. This combination has become a new first-line treatment option for adult patients with advanced bladder cancer; clinical trials demonstrated that it significantly prolonged progression-free survival and overall survival compared to standard chemotherapy, and it has been available through the UK's National Health Service (NHS) since August 21.

BeOne Medicines' (Bristol Myers Squibb's) PD-1 inhibitor, Tevimbra, has gained further recognition from European regulators: the European Commission approved its use in combination with platinum-containing chemotherapy as neoadjuvant (preoperative) treatment for adult patients with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence, followed by adjuvant monotherapy with Tevimbra after surgery. This marks Tevimbra's fifth lung cancer indication in the EU; previously, in July, the EU also approved its use in combination with gemcitabine and cisplatin as first-line treatment for adult patients with metastatic or recurrent nasopharyngeal carcinoma (NPC).

Other Therapeutic Areas: Approval Dynamics and Label Adjustments

In the field of Alzheimer's disease treatment, following the approval of the subcutaneous formulation of Eisai and Biogen's Leqembi for maintenance therapy, the companies have initiated a rolling submission to the FDA seeking approval for Leqembi Iqlik, an autoinjector for weekly initial dosing. This autoinjector serves as an alternative to the biweekly 10mg/kg intravenous administration; the subcutaneous formulation for the maintenance phase is a single-dose prefilled autoinjector containing 360mg, with an administration time of approximately 15 seconds. It is expected to allow patients and caregivers to administer treatment at home from the initial phase through the maintenance phase.

Meanwhile, the FDA has updated Leqembi's prescribing information, recommending that patients undergo an MRI scan before the third infusion to identify amyloid-related imaging abnormalities with edema (ARIA-E) - a revision from the previous recommendation of scans before the fifth, seventh, and fourteenth infusions. This adjustment stems from the analysis of 6 deaths reported during early treatment.

In the rare disease space, SpringWorks Therapeutics' Ogsiveo has been approved in Europe, becoming the world's first drug for the treatment of desmoplastic tumors. It is indicated for adult patients with progressive desmoplastic tumors requiring systemic treatment. Data from the phase III DeFi study showed that Ogsiveo reduced the risk of disease progression by 71% compared to placebo, and also demonstrated superiority in endpoints such as objective response rate and patient-reported outcomes. The drug had already obtained approval in the U.S. in 2023.

Shilpa Medicare's norursodeoxycholic acid (NorUDCA) tablets have received the world's first approval for the treatment of non-alcoholic fatty liver disease (NAFLD) in India. Through its dual mechanisms of anti-inflammation and bile acid regulation, NorUDCA prevents NAFLD from progressing to cirrhosis, liver failure, and non-alcoholic steatohepatitis (NASH). Globally, approximately 1.2 billion people are affected by NAFLD, with around 188 million patients in India.

Regulatory Adjustments and Challenges: Approval Delays and Safety Measures

Some drugs are facing approval delays. The FDA has requested additional information for Agios' Pyrukynd, which is under review for the treatment of thalassemia - including the submission of a Risk Evaluation and Mitigation Strategy (REMS) to reduce the risk of hepatocellular injury. This constitutes a major amendment, leading the FDA to extend the decision date from September 7 to December 7. Pyrukynd is intended for the treatment of adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) α- or β-thalassemia.

Chugai Pharmaceutical (a subsidiary of Roche) has implemented new safety measures for its Duchenne muscular dystrophy gene therapy, Elevidys, in Japan, adding acute liver failure as a serious adverse reaction to the drug's label. Patients are required to undergo testing for liver dysfunction and liver failure before and after administration. This follows the deaths of two ambulatory patients in the U.S. after receiving the therapy.

Furthermore, the FDA has adjusted safety monitoring requirements for the labels of certain drugs. For example, the REMS label for Travere Therapeutics' IgA nephropathy drug, Filspari, has been updated: the frequency of liver function monitoring has been changed from monthly to once every three months after the start of treatment, and the previous embryofetal toxicity (EFT) monitoring requirement has been removed.