Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Between 2011 and 2020, 410 new molecular entities were approved by the FDA, reflecting a 6.5% growth in the US. Globally, expedited approval programs have been crucial for anticancer drugs, with 9-75% receiving such designations in Europe, and China implementing similar pathways to enhance pharmaceutical development.
Read full storyIn 2024, the biotech sector has seen 47 M&A and IPO deals totaling $53 billion, with 15 exceeding $1 billion. Notably, cancer, rare diseases, and cell therapies are key areas attracting investment.
A systematic review and meta-analysis reveals the global prevalence of peripheral neuropathy in cancer patients post-chemotherapy. This study highlights the significant impact of chemotherapy on nerve health, emphasizing the need for better management strategies.
Eikon Therapeutics raised $381 million in its IPO to advance its lead cancer immunotherapy program currently in clinical trials. The offering included 21.2 million shares priced at $18 each, reflecting strong investor interest in the biotech sector.
Novartis has cut six early-stage cancer candidates while adding two to refine its oncology strategy. CEO Vas Narasimhan emphasized ongoing investment in the early-stage pipeline and a focus on deals under $2 billion.
Pharma Times highlights the shift towards patient-centric oncology trials, emphasizing the use of data-driven insights to enhance cancer care. This approach aims to improve patient outcomes by integrating their experiences and preferences into trial designs.
Current policy debates question the alignment of the Orphan Drug Act’s incentives with patient needs, as rising costs and access issues persist. The proposed ORPHAN Cures Act aims to enhance drug indications for rare diseases, potentially improving treatment access and outcomes.
Congress has passed a landmark bill aimed at protecting patient access to specialty pharmacies, crucial for managing complex health conditions such as cancer and rare diseases. This legislation is expected to stabilize the specialty pharmacy infrastructure, enhancing innovation and patient outcomes.
Daiichi Sankyo has decided to discontinue the development of DS-9606, its next-generation antibody-drug conjugate intended for cancer treatment. This decision reflects a strategic shift in their oncology pipeline, impacting future funding and resource allocation.
The National Cancer Institute (NCI) is seeking public comment on a proposed data submission request form for the Genomic Data Commons (GDC). This initiative is part of compliance with the Paperwork Reduction Act, allowing stakeholders to provide input before submission to the Office of Management and Budget.
The CDC is actively conducting and supporting studies aimed at reducing cancer incidence and eliminating health disparities. This initiative underscores the agency's commitment to addressing inequities in cancer care and prevention.
The CDC is seeking public comment on a proposed information collection project aimed at evaluating the impact of Training and Technical Assistance programs for the National Comprehensive Cancer Control Program (NCCCP). This initiative aligns with the Paperwork Reduction Act of 1995 to enhance government information utility.
Aktis Oncology targets a valuation of up to $945.4 million in its upsized IPO, with Eli Lilly as an anchor investor, highlighting strong investor interest in biotech. This partnership builds on their 2024 collaboration to develop tumor-targeting radiopharmaceuticals.
The CDC's NIOSH released the First Periodic Review of Scientific and Medical Evidence Related to Cancer for the World Trade Center Health Program, summarizing current findings on cancer related to exposures from the September 11 attacks. This review highlights the ongoing research into the health impacts of the WTC disaster.
The Annual Report to the Nation on the Status of Cancer reveals a continued decline in cancer death rates for both men and women from 2003 to 2007. Additionally, the overall rate of new cancer diagnoses decreased by nearly 1% per year during this period.
The number of cancer survivors in the U.S. has reached 11.7 million, up from 3 million in 1971 and 9.8 million in 2001. This growth highlights the advancements in cancer treatment and survivorship over the decades.
CDC reports that smokers of certain U.S. cigarette brands are exposed to higher levels of cancer-causing chemicals, specifically tobacco-specific nitrosamines. This finding highlights the need for increased awareness and potential regulatory actions regarding tobacco products.
The Annual Report to the Nation reveals a significant decline in cancer incidence and death rates for the first time since 1998, with notable variations in lung cancer trends across states. This trend is largely attributed to reductions in some of the most prevalent cancer types.
The Annual Report to the Nation reveals that the cancer death rate in the U.S. has declined at double the previous rate, highlighting significant progress in cancer treatment and prevention. The report also includes a special feature on cancer impacts among American Indians and Alaska Natives.
The CDC's 2007 Cancer Conference will address future challenges in cancer prevention and early detection through various presentations and topic sessions. This meeting aims to foster discussions on critical issues in the field.
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