Signs are seen outside the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, on August 29, 2020.
Andrew Kelly | Reuters
A version of this article first appeared in CNBC's Healthy Returns newsletter, which brings the latest health news straight to your inbox. Subscribe here to receive future editions.
The U.S. Food and Drug Administration closed 2024 with 50 new drug approvals, capping a wave of long-awaited treatments.
This emerges from the agency's official tally of new treatments that have not yet been approved or marketed in the United States. The total number of approved drugs fell slightly from 55 in 2023, but there were several firsts and other notable drugs that the agency gave the green light last year.
This includes the first-ever treatment for metabolic dysfunction-associated steatohepatitis (MASH), a common and potentially fatal liver disease that affects millions of people worldwide. The drug Rezdiffra comes from Madrigal Pharmaceuticalswhich is now succeeding in a disease area where several large companies have failed or are still trying to break into.
The FDA also approved the first two treatments for a rare progressive genetic disorder called Niemann-Pick disease type C. The condition causes damage to the nervous system over time because the body cannot properly transport cholesterol and other fatty substances into cells, leading to a buildup of these substances in the body.
The authority granted the first approval for a drug called Miplyffa Zevra Therapeuticswhich reached the finish line on its second attempt after a previous rejection in 2021. Aqneursa, a private company owned by IntraBio, also received FDA approval for the treatment of Niemann-Pick disease type C.
The FDA also approved several drugs last year that aim to treat known diseases in new ways.
For example, Bristol Myers SquibbCobenfy is the first novel treatment approved for schizophrenia in decades. It is the first drug in a new class that does not directly block dopamine to improve symptoms.
Cobenfy drug from Bristol Myers Squibb
Courtesy: Bristol Myers Squibb
PfizerThe new drug for hemophilia A or B, genetic blood clotting disorders, is also the first to target a specific protein in the blood clotting process. The treatment, Hympavzi, is also the first U.S.-approved hemophilia drug specifically delivered using a prefilled autoinjection pen.
MerckWinrevair was the first drug to target the root cause of pulmonary arterial hypertension, a progressive and life-threatening lung disease. Other medications available only help relieve symptoms.
The FDA has now granted approval AmgenImdelltra therapy is used as a second- or second-line therapy for people with advanced small cell lung cancer. It is the first and only so-called T cell engager therapy approved for this deadly form of lung cancer.
Amgen's drug is specifically designed to redirect the immune system's T cells to recognize and kill cancer cells.
Other notable FDA approvals include: Eli Lillyis the long-awaited drug for early-onset Alzheimer's disease that faced several obstacles on its path to market. The treatment, Kisunla, expands the limited treatment options in the United States for the disease that causes mental degeneration.
Notably, many of the FDA approvals involved products from a mix of little-known public and private companies such as Allecra Therapeutics, Iovance Biotherapeutics and BridgeBio Pharma. This signals that smaller biotech companies are more willing to bring their products to market themselves rather than partnering or being acquired by a large pharmaceutical company.
Feel free to send tips, suggestions, story ideas and data to Annika at annikakim.constantino@nbcuni.com.
Latest technology in healthcare: Transcarent takes Accolade private for $621 million
Digital health startup Transcarent announced the acquisition on Wednesday accolade in a deal worth around $621 million.
Transcarent, which earned a spot on CNBC's Disruptor 50 list last year, offers at-risk pricing models to self-insured employers to help their employees quickly access care and manage benefits. As of May, the company had raised about $450 million at a valuation of $2.2 billion.
Accolade provides care, navigation and advocacy services. The company went public during the coronavirus pandemic in 2020 as investors began pouring billions of dollars into digital health, but the stock has largely been in free fall since then. Accolade is the latest in a string of digital health companies to exit public markets as the sector readjusts post-Covid.
Transcarent will acquire Accolade for $7.03 per share in cash, a premium of about 110% to the stock's closing price on Tuesday, Transcarent said. The transaction is expected to close in the next quarter but is subject to shareholder and regulatory approval.
“By combining Transcarent's complex care experience with Accolade's people and 16 years of health data, we will provide people with a more personalized healthcare experience while improving outcomes and reducing costs,” Accolade CEO Rajeev Singh said in a statement on Wednesday .
Evercore acted as financial advisor to Transcarent on the transaction. Morgan Stanley advised Accolade.
Glen Tullman, CEO of Transcarent, is familiar with eye-catching deals in the digital health sector. Tullman previously led Livongo, which was acquired by virtual care provider Teladoc in a 2020 deal that valued the company at $18.5 billion.
When Teladoc acquired Livongo, the companies had a combined enterprise value of $37 billion. Teladoc's market cap is currently around $1.7 billion.
“I think since Livongo the power of digital health care has slowed down a bit,” Tullman said in an interview with CNBC on Wednesday. “We’re now creating an experience that people not only like, but actually love.”
Tullman said Transcarent and Accolade had “very similar visions.” Accolade, for example, has deep expertise in medical reviews, primary care and patient advocacy, which he said Transcarent's customer base was looking for.
“I think it was a perfect fit,” Tullman said.
Read the full press release here.
Feel free to send tips, suggestions, story ideas and data to Ashley at ashley.caroot@nbcuni.com.
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