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FDA Advances Additional Activities to Prevent Drug Overdoses and Reduce Death

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Addressing the drug overdose crisis and substance use disorder (SUD) is an issue of great concern for our nation and remains a top public health priority for the U.S. Food and Drug Administration. Upon returning to the FDA as Commissioner, I expressed my commitment to respond to all aspects of this ongoing crisis. As an agency focused on protecting public health, combatting this evolving emergency is an issue of particular urgency for us. Of note, a public health emergency, as a consequence of the opioid crisis, was declared by the U.S. Department of Health and Human Services in October 2017 and renewed on December 22, 2022.  

In August 2022, I announced the FDA’s Overdose Prevention Framework – our expanded vision to undertake creative actions with an impact not only on preventing drug overdoses and reducing deaths from opioids and other substances, but also ameliorating the tremendous toll of addiction on American individuals and families. As part of that announcement, I shared that the agency would commission an independent external opioid-related activities review. That review was conducted by a team of subject matter experts affiliated with The Ohio State University who are familiar with the 2017 National Academies for Sciences, Engineering, and Medicine’s (NASEM) recommendations, a project initiated on my watch, and also related opioid prescribing issues. The review analyzed the FDA’s implementation of the NASEM recommendations along with key regulatory policies and decisions, including labeling. 

Today, we are sharing that report, which provides lessons learned, recognizes the work already being done by the FDA, and makes the following three overarching recommendations outlined for the agency to take action: 

  • Continue efforts to comprehensively implement the recommendations in the 2017 NASEM Report, including evaluating scientifically-sound, inclusive study designs to inform a systems approach for regulatory decision-making that incorporates public health considerations.  
  • Consider seeking from Congress certain additional authorities regarding opioid analgesic approvals and review of the advertising and promotion for such products, as well as additional resources to implement such authorities to strengthen oversight of prescription opioid analgesics.
  • Be as transparent as possible regarding decision-making for opioid analgesics, as increased transparency can encourage appropriate uses of prescription opioid analgesics, promote innovation in pain management and prevention of opioid use disorder, and enhance public trust.

We have reviewed these recommendations and while we are thoughtfully considering our actions to implement them, I am encouraged that the agency is already moving forward on multiple workstreams, while also adapting to the changing epidemiology of the ongoing public health emergency. New challenges confronting us include much more multidrug use and troubling distribution of powerful chemically-synthesized drugs. 

While our response is centered on opioid regulation by the Center for Drug Evaluation and Research (CDER), all elements of the agency are involved as we work with digital applications of substance use disorder treatment, the inappropriate intermixing of prescription drugs—like certain veterinary medicines—in the illicit market, enforcement activities in the domain of the Office of Regulatory Affairs and the intersection of nicotine addiction with use of other addictive substances as the Center for Tobacco Products oversees tobacco products.  

The activities outlined below are a sample of the broad and energetic effort across the agency to combat this emergency: 

First, we’re taking steps to strengthen evidence generation within the agency and with multiple partner organizations in order to better inform our response to the opioid crisis. The FDA has invested significant resources to develop a data-driven, national-level simulation model of the opioids system, named SOURCE. We will continue to enhance systems modeling approaches, including SOURCE, to enable identification of evolving trends and gaps in our interventions.

Of particular interest is deepening our understanding of the long-term efficacy of opioid analgesics. To clarify the current state of this research and knowledge gap, we will hold an advisory committee meeting in April 2023. It will focus on clinical trial paradigms, including an enriched enrollment randomized withdrawal trial design to evaluate long term efficacy and the risk of opioid-induced hyperalgesia. This is part of a postmarketing requirement we issued to application holders of new drug applications for extended release and long-acting opioid analgesics. We appreciate the revised 2022 CDC Clinical Practice Guideline for Prescribing Opioids for Pain that once again pointed out the paucity of evidence on the potential benefits of long-term opioid use, raising questions for our consideration about the labeling of these drugs.

I had the opportunity to contribute to the planning in 2015-16 that eventually led to the NIH’s HEAL Initiative, which has multiple ongoing studies evaluating the clinical epidemiology, health services and interventions in clinical practice. As this extensive network conducts its work, we anticipate a steady stream of information that will inform our policies.

This information, along with other scientific evidence and insight, will aid our science-based evaluations of the benefit-risk assessment of opioid products, and help ensure that the benefit-risk assessment considers not only the outcomes of prescription opioids when used as prescribed but also the public health effects of inappropriate use.

Next, the report suggests the agency should consider seeking additional authorities through Congress. We know that having fit for purpose regulatory authority is key to addressing opioid use, misuse and abuse. As referenced in the agency’s Overdose Prevention Framework, we are exploring the need for potential new authorities for opioid approval standards, including whether drug developers seeking approval for the marketing of new opioid analgesics should be required to demonstrate that their products offer material safety advantages over existing approved opioid analgesics. 

For many years, the FDA has based decisions on whether to approve drugs, including opioids, on a framework for the assessment of the benefits and risks of a drug. That approach is the authority Congress mandated for us. When it comes to opioids, we also take into account public health factors such as the impact on family and others when reaching a decision on whether to approve a drug. Currently, the FDA lacks the statutory authority to require that drug developers seeking approval for the marketing of new opioid products demonstrate that their products offer material safety advantages (such as a reduction in abuse potential or a reduction in respiratory depression) over existing approved opioids. By enabling the FDA to refuse to approve new opioid analgesics that do not offer material safety advantages over already-approved opioids there is potential to, over time, improve the safety of opioid analgesics available to treat severe acute and chronic pain or drive research towards non-opioid alternatives to relieve pain. We stand ready to work with Congress on an improved regulatory tool for the agency.  

As we examine the need for new authorities, we will continue to explore ways to use our current authorities, not only to regulate opioids more effectively, but also to improve the pipeline of non-addictive treatments for pain.  While this has been a goal for many years now, the industry has been disappointingly unable to develop these much-needed alternatives.  

Lastly, continuously working to earn and retain the public’s trust is not only important to our work, but also critical to our mission to protect public health. The report correctly highlights the importance of transparency in our decisions regarding opioid analgesics. 

An area where we believe we can increase transparency is through further improvements of our advisory committee meetings. Our commitment to explore more effective use of our advisory committees to increase transparency regarding opioid analgesics will be a part of a broader internal effort to evaluate how to make the best use of all of our advisory committees and ensure agency decisions are informed by input from knowledgeable external advisors and the public and the decisions are shared publicly. Advisory committees play a crucial role in regulatory decision-making and publicly clarify the FDA’s expectations and the views of experts in the field while giving the public a chance for direct input. Although the airing of opinions at advisory committees, like democracy, can be messy, by improving transparency, better public understanding of the FDA’s decision-making process will lead to more confidence in it.

Within each of the overarching recommendations outlined are additional, more specific recommendations that we will continue to address and prioritize through our existing authorities and current activities within CDER and beyond. 

I look forward to continuing to work across the agency to advance solutions that encourage appropriate prescribing, promote innovation in pain management, prioritize overdose prevention, reduce opioid and other substance use disorders, and champion effective treatment and support for those with SUD. We recognize that in the face of this ongoing emergency, we must continuously assess and improve our efforts and I appreciate the commitment of the FDA’s center directors and staff in this continuing effort while also dealing with other challenges that impact public health.

Related Information

Source: FDA

FDA CDC News

FDA Approval of Nonsteroidal Treatment for Duchenne Muscular Dystrophy

Breaking news! FDA approves Duvyzat for Duchenne Muscular Dystrophy, a nonsteroidal treatment providing hope for patients and families. #DMD #FDAApproval

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Breaking news in the medical world! The U.S. Food and Drug Administration has just approved Duvyzat (givinostat), an oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older. Duvyzat is making history as the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. This is a significant milestone in the treatment of this devastating disease.

DMD, being the most common childhood form of muscular dystrophy, primarily affects males. It is a neurological disorder that leads to progressive muscle weakness due to a lack of dystrophin, a muscle protein. Over time, the muscles deteriorate, causing difficulties with walking, muscle strength, and eventually leading to breathing problems and early death. However, with advancements in treatment, the life expectancy for individuals with DMD has been steadily increasing, with some patients surviving beyond 30 years.

Duvyzat is a histone deacetylase (HDAC) inhibitor that targets pathogenic processes to reduce inflammation and loss of muscle in patients with DMD. Its efficacy for the treatment of DMD was evaluated in an 18-month phase 3 study involving a randomized, double-blind, placebo-controlled trial. The primary endpoint of the study was the change in muscle function, measured by the time it took patients to climb four stairs. Patients treated with Duvyzat showed a statistically significant reduction in the decline of muscle function compared to placebo.

Another measure of efficacy was the change in physical function assessed by the North Star Ambulatory Assessment (NSAA), a scale commonly used to rate motor function in boys with DMD who can still walk. Patients treated with Duvyzat experienced less worsening in their NSAA scores after 18 months, compared to those on placebo.

Like any medication, Duvyzat does come with some potential side effects. The most common ones reported were diarrhea, abdominal pain, a decrease in platelets, nausea/vomiting, an increase in triglycerides, and fever. It’s important for healthcare providers to evaluate a patient’s platelet counts and triglyceride levels before prescribing Duvyzat. Patients with low platelet counts should not take the drug. Monitoring of platelet counts and triglycerides throughout treatment will help determine if any dosage adjustments are needed.

Healthcare professionals should also be aware that Duvyzat may cause QTc prolongation, which can increase the risk of irregular heartbeats. It’s crucial for patients taking other medications known to cause QTc prolongation or with certain types of heart disease to avoid taking Duvyzat.

The recommended dosage of Duvyzat is determined by the patient’s body weight, and it should be taken orally twice daily with food.

The approval of Duvyzat was granted to Italfarmaco S.p.A., and it received priority review, fast-track designation, as well as orphan drug and rare pediatric disease designations from the FDA. This demonstrates the agency’s commitment to advancing the development of new therapies for DMD and its recognition of the urgent need for effective treatments.

This milestone approval represents hope and progress for individuals and families affected by DMD. It provides another treatment option to help reduce the burden of this progressive and debilitating disease, regardless of the specific genetic mutation. With continued advancements in medical research and the dedication of organizations like the FDA, we are moving closer to a future where individuals with DMD can lead healthier lives.

As always, it’s important to consult with healthcare professionals for more information and guidance regarding Duvyzat and its suitability for individual cases. Let’s celebrate this achievement in medical science and look forward to more groundbreaking developments in the treatment of Duchenne Muscular Dystrophy.

Source: FDA

Related Information

  • Duchenne muscular dystrophy – About the Disease – Genetic and Rare Diseases Information Center
  • The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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Understanding RSV: Symptoms, Treatment, and Prevention

RSV: a viral respiratory infection affecting children and adults. Learn about symptoms, treatment, and prevention for a healthier future.

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Photo by Vlada Karpovich on Pexels.com

RSV, short for Respiratory Syncytial Virus, is a common viral infection that primarily affects the respiratory system, particularly young children. While RSV infections are usually mild, they can pose a greater risk to infants, older adults, and individuals with weakened immune systems. If you or a loved one are facing RSV, it can be overwhelming to know what to do next. That’s why it’s important to know the symptoms, treatment options, and preventative measures available to you. By being informed and taking action, you can give yourself and those around you hope and guidance during this difficult time. So let’s explore what you can do to fight RSV and emerge victorious.

Symptoms and Diagnosis:
RSV presents symptoms resembling a cold, including cough, runny nose, fever, and occasionally, difficulty breathing. Infants may exhibit irritability, decreased appetite, and lethargy. Diagnosing RSV usually involves a medical professional evaluating symptoms, conducting a physical examination, and, if necessary, performing laboratory tests to confirm the presence of the virus.

Treatment and Management:
In most cases, RSV infections can be managed at home with supportive care. This includes ensuring proper hydration, maintaining a comfortable environment, and using over-the-counter medications to alleviate symptoms under a doctor’s guidance. However, for high-risk individuals or severe cases, hospitalization may be required for closer monitoring and specialized treatment, such as oxygen therapy or intravenous fluids.

Prevention is Key:
Preventing the spread of RSV is crucial, especially for vulnerable populations. Practicing good hygiene, such as frequent handwashing, covering coughs and sneezes, and avoiding close contact with infected individuals, can significantly reduce the risk of transmission. Additionally, promoting a clean and sanitized environment, particularly in daycare centers and schools, can help curb the spread of the virus.

Hopeful Outlook:
While RSV can be concerning, it’s important to remember that most cases resolve on their own with time and supportive care. In fact, the majority of children infected with RSV recover fully without complications. By following preventive measures, seeking medical attention when needed, and staying informed about the latest developments in RSV research and treatment, we can approach this viral infection with hope and confidence.

Respiratory Syncytial Virus (RSV) is a common viral infection that primarily affects young children, causing cold-like symptoms and occasionally resulting in more severe respiratory distress. However, with proper care and attention, RSV can be managed effectively. By understanding the symptoms, seeking medical help when necessary, and adopting preventive measures, we can protect ourselves and our loved ones from the impact of RSV and look forward to brighter days ahead.

You can find more information about RSV, including its treatment and prevention, by visiting the CDC website. https://www.cdc.gov/rsv/index.html

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FDA Approves First Therapy for Rare Type of Non-Cancerous Tumors

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Today, the U.S. Food and Drug Administration approved Ogsiveo (nirogacestat) tablets for adult patients with progressing desmoid tumors who require systemic treatment. Ogsiveo is the first drug to be approved for the treatment of patients with desmoid tumors, a rare subtype of soft tissue sarcomas.

Desmoid tumors are non-cancerous but can be locally aggressive. The tumors may invade into surrounding structures and organs, resulting in pain, issues with being able to move, and decreased quality of life. Although surgical removal has historically been the treatment of choice, there is a high risk that the tumor will return or that other health challenges will occur after removal; therefore, systemic therapies (cancer treatment targeting the entire body) are being increasingly evaluated in clinical trials. 

“The FDA continues to address unmet medical need and advance the development of safe and effective therapies for the millions of Americans whose lives are affected by rare tumors,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Desmoid tumors are rare tumors that can lead to severe pain and disability. Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

The effectiveness of Ogsiveo was evaluated in an international, multicenter, randomized, double-blind, placebo-controlled trial in 142 adult patients with progressing desmoid tumors not amenable to surgery. Patients were randomized to receive 150 milligrams (mg) of Ogsiveo or placebo orally, twice daily, until disease progression or unacceptable toxicity. The main efficacy outcome measure was progression-free survival (the length of time after the start of treatment for which a person is alive and their cancer does not grow or spread). Objective response rate (a measure of tumor shrinkage) was an additional efficacy outcome measure. 

The pivotal clinical trial demonstrated that Ogsiveo provided clinically meaningful and statistically significant improvement in progression-free survival compared to placebo. Additionally, the objective response rate was also statistically different between the two arms with a response rate of 41% in the Ogsiveo arm and 8% in the placebo arm. The progression-free survival results were also supported by an assessment of patient-reported pain favoring the Ogsiveo arm. 

The most common side effects seen in at least 15% of the patients in the trial were diarrhea, ovarian toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal pain, cough, alopecia, upper respiratory tract infection and dyspnea. 

Ogsiveo was granted Priority Review under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition compared to available therapies. Ogsiveo also received FDA Fast Track and Breakthrough Therapy designations for the indication noted above, as well as Orphan-Drug designation for treatment of desmoid tumor (aggressive fibromatosis). Orphan-drug designation provides incentives to assist and encourage drug development for rare diseases.

The FDA granted the approval of Ogsiveo to SpringWorks Therapeutics Inc.

Source: FDA

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