2024 was a busy and eventful year for pharma; year-to-date the FDA granted 46 new drug approvals. To look forward, we sometimes need to take stock of our wins and missteps and analyze the current landscape. While I couldn't cover every development, such as the significant impact of GLP-1s or the progress made in the fight against cancer, here’s a curated selection of pharma stories that caught my eye in 2024.
Mpox: Mpox, formerly called monkeypox, returned in 2024 after the 2022 viral outbreak got under control. For 2024 (as of December) there were almost 26,000 reported cases in Africa with at least 100 confirmed deaths. The disease has not widely spread globally; seven countries have reported a single case including the U.S., Canada, Germany, Sweden, India, and Thailand, with five cases in the U.K.
The World Health Organization declared Mpox a global emergency in July, calling for vaccine donations for African countries. Nations, organizations, and manufacturers responded (perhaps not as strongly as hoped), and vaccinations are underway in some areas, with plans and strategies being determined in others. By November, the Democratic Republic of the Congo, one of the harder-hit countries, had received 100,000 doses and were immunizing. An additional 1,678,000 doses were ready for shipment to Africa in early December. Despite the strides made in vaccine accessibility, the growing wave of vaccine skepticism continues to be a major concern, particularly with contagious, yet preventable diseases like mpox.
Sickle Cell Disease: In September, the first U.S. patient was treated with Lyfgenia, a gene therapy for sickle cell disease, after a December 2023 FDA approval. Kendric Cromer, 12 years old, made history undergoing this multi-step gene therapy process. It was not easy for Kendric, and it was pricey, but the goal is for this treatment to cure his disease.
Presuming it works and there are no devastating or unexpected side effects, others will likely follow his path. Although this gene therapy in its current form is a major pharmaceutical advancement, it will not be scaled up to treat the majority of patients, given constraints on human and financial resources. Yet, it still can improve the health and quality of life for some of the 100,000 people in the U.S. with sickle cell disease. Later in September, Pfizer unexpectedly withdrew its sickle cell disease treatment, Oxbryta (voxelotor), from the market after determining that the risks outweighed the benefits. For the sickle cell community, this year has been a hit-and-miss. Treatment remains complex, with limited options available.
Hemophilia: In another win for patients and pharmaceutical advancements, Pfizer received two FDA approvals in 2024 for hemophilia treatments: Beqvez and Hympavzi. Approved this spring, the gene therapy Beqvez can treat some adults with severe and moderately severe hemophilia B. The anti-tissue pathway inhibitor Hympavzi was approved for some patients 12 and older with Hemophilia A or B, to limit bleeding episodes. This prophylactic treatment is user-friendly and is given weekly with an auto-injector pen.
These are both welcome advancements in hemophilia, as there has been a dearth of options in recent years. I’m especially thrilled to see Hympavzi gain approval as it was part of my development portfolio in my role as Global President of Rare Disease at Pfizer from 2016 to 2017.
The wheels of progress move slowly, and success is never guaranteed, so we need to celebrate these wins.
Alzheimer’s disease: Another area in medicine where we desperately need better treatments is Alzheimer’s disease. Sometimes we take one step forward and one step back, that was what happened with Aduhelm, which was discontinued this year, in order to better allocate resources. On a more positive note, in July, the FDA approved Kisunla to treat early Alzheimer’s disease. This disease-modifying monoclonal antibody therapy is approved for those with mild cognitive impairment or mild dementia due to Alzheimer’s disease. In a world with limited treatment options, it’s a start.
Schizophrenia: In September, those with schizophrenia received a new treatment option. Cobenfy is a new approach to antipsychotic treatment, targeting cholinergic receptors instead of the traditional dopamine receptors. Given that schizophrenia is one of the top 15 causes of disability, finding the right treatment for these individuals is vital and it’s refreshing to have a novel option on the market.
Speaking of new options, 2024 brought innovation in other treatment approaches as well.
Biosimilars: There are now 10 FDA-approved biosimilar alternatives to Humira. In spite of cost savings and a few biosimilars with interchangeability status, there’s still an uphill adoption struggle. I dove into why these drugs are battling for market share and hint at what’s to come in 2025. Read more here.
Rare diseases and gene editing: While individual rare diseases affect fewer than 5 or 6 people per 10,000, the toll adds up when considering that there are at least 10,000 rare diseases in existence and together they affect over 400 million people globally. The majority (72%) of rare diseases have genetic causes, so not surprisingly, scientists are looking into gene editing options. There’s no greater excitement in this arena than the idea of stopping a disease before birth. Researchers are investigating options to do this through in-utero gene editing. Of course, this has challenges and ethics concerns, which you can read more about here.
mRNA vaccines and expedited vaccine creation: The ease by which mRNA vaccines for COVID-19 have become a plug-and-play option is truly incredible when you realize that this platform was still in the clinic in 2019. While the pandemic was devastating in many ways, one bright spot is how the world (governments, corporations, and nonprofit organizations) came together to expedite vaccine creation safely and effectively. I previously wrote about how the pandemic advanced vaccine development here.
We have much to look forward to as we head into the new year. Between expected approvals for new drugs and building on the progress our industry has made this year, are there any specific approvals you’re anticipating?
Let’s continue the conversation.
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