FDA approves new drug for hemophilia

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Updated Apr 3, 2025 | 06:54 AM IST

FDA Approves Qfitlia For Hemophilia Patients

SummaryThe approval came on the basis of two randomized clinical trials that had 177 adult and pediatric male patients with either hemophilia A or hemophilia B enrolled.

The United States Food and Drug Administration has recently approved Qfitila (fitusiran) for routine prophylaxis to prevent or to reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with hemophilia. It works on those with hemophilia A or hemophilia B, with or without factor VII or IX inhibitors which neutralizes antibodies.

While subcutaneous Qfitilia does not replace the missing coagulation factor VII or IX, it reduces the amount of protein antithrombin, which leads to an increase in thrombin. It is an enzyme critical for blood clotting. The dosing starts at once every two months and it is also based on the FDA-cleared Innovance Antithrombin companion diagnostic test.

However, as of now, there is no fixed doses of Qfitila is not approved mainly due the risk excessive clotting. It has also a boxed warning for thrombotic events and gallbladder disease.

One of the most common side effects reported were viral infection, nasopharyngitis, and bacterial infection.

How Was This Approved?

The approval came on the basis of two randomized clinical trials that had 177 adult and pediatric male patients with either hemophilia A or hemophilia B enrolled. Among the participants with inhibitors who received the antithrombin-based dosing regimen of Qfitlia, there was a 73% reduction in the estimated annualized bleeding rate. This was seen in comparison with those who received on-demand treatment with bypassing agents.

Among the participants without inhibitors receiving the antithrombin-based dosing regimen of Afitlia, there was a 71% reduction in the estimated annualized bleeding rate versus that seen in those who received on-demand treatment with clotting factor concentrates.

"Today's approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options," Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA Center for Drug Evaluation and Research, said in a statement.

What Is Hemophilia?

As per the Centers for Disease Control and Orevention (CDC) it is an inherited bleeding disorder in which blood does not clot properly. It can lead to spontaneous bleeding as well as bleeding following injuries or surgeries. Blood usually has proteins called clotting factors that can help one to stop bleeding. However, people with hemophilia have low levels of either factor VII or factor IX.

Hemophilia could also result in bleeding within joints which can then lead to chronic joint disease and pain. It could lead to bleeding in head and in brain, causing long term problems such as seizures or paralysis. In severe cases it can also lead to death if the bleeding cannot be stopped.

The most common types of hemophilia are inherited, thus it is a condition that is present from birth. There are rare cases where hemophilia happens later in life, which is known as acquired hemophilia.

Difference between Hemophilia A and Hemophilia B

As per the National Bleeding Disorders Foundation, hemophilia A, also called factor VIII (8) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII (FVIII), a clotting protein. Although it is passed down from parents to children, about 1/3 of cases found have no previous family history.

Hemophilia B, also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder caused by missing or defective factor IX, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene.

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Flourishing Does Not Mean Happiness, Finds Study

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Updated May 2, 2025 | 07:00 AM IST

Flourishing Doesn't Mean Happiness, Youth Is Struggling, Finds Study

SummaryA global study shows flourishing goes beyond happiness, with Indonesia leading. Younger adults struggle most, and richer nations lag in meaning and relationships despite financial security and health.

A new study titled The Global Flourishing Study, conducted by researchers from Baylor and Harvard universities have thrown light on the meaning of "flourishing" and how it does not mean being happy. The study has looked at how some countries are doing significantly better in terms of the broader sense of well-being than other countries. The study is based on responses from over 207,000 participants across 22 countries and Hong Kong. The study also assessed six core aspects of flourishing, which includes happiness, health, meaning, character, relationships, and financial stability.

What Does Being "Flourished" Really Mean?

While there are traditional happiness indexes that measures an individual's or a group's subjective well-being or happiness, the study about being "flourished" includes not just how happy people feel, but also their health. The study looks at whether people are leading a healthy life, whether they are mentally well and feel like they do have a purpose in life. The study also takes in account for strong relationships, acting with character and feeling financially secure.

The participants of this study were asked to respond to 12 key questions. They were aimed at measuring these components. The questions varied from "How satisfied are you with life?" to "Do you worry about meeting monthly expenses?"

Dr. Byron Johnson of Baylor University, who led the study, said the scale of the research is what makes it especially unique. “We are following 207,000 participants around the world in over 40 languages. This gives a voice to approximately 64% of the world’s population,” he explained.

Indonesia Leads, But Traditionally 'Happy' Nations Lag

The results too have been unexpected, unlike what we seen in the happiness indexes. Countries which were often seen as happy in global rankings did not perform as well when it came to flourishing. Indonesia, however, topped the list, followed by Mexico and the Philippines. However, nations like Sweden and the United States, which are usually high ranking in the World Happiness Report, were found to be average in terms of flourishing.

Interestingly, some of the most flourishing countries, such as Nigeria and Indonesia, did not make it to the top 20 in the World Happiness Report, indicating a crucial distinction between simple happiness and a deeper sense of well-being.

Youth Is Struggling

One of the most concerning findings of the study was that flourishing tends to increase with age. According to Dr. Tyler VanderWeele of the Harvard T.H. Chan School of Public Health, young people across most countries reported the lowest levels of flourishing, often citing a lack of purpose.

"In most countries, the youngest individuals are the ones struggling the most,” VanderWeele noted. However, there were exceptions—Tanzania and Poland showed relatively better flourishing scores among their younger populations.

The researchers suggest that in more developed countries, increased competition, job stress, and social comparison may be contributing to this generational dip. “Young people are telling us something is wrong,” added Felix Cheung, a co-author of a separate study in the World Happiness Report.

Rich, Not Flourishing

While wealthier countries usually scored higher in the financial security section, they lacked in the sections where they were asked questions on meaning and relationships.

This gap has raised questions about modern development. “How can we carry out economic growth without compromising meaning and relationships?” VanderWeele asked.

In contrast, countries with fewer economic resources but stronger community bonds and clearer cultural or spiritual values often performed better on overall flourishing metrics.

According to researchers, people can take active steps to improve their own flourishing. VanderWeele shared how one participant, after reflecting on the study’s 12 questions, decided to volunteer to find greater purpose. “Flourishing can be guided by reflection,” he said.

Still, systemic issues like conflict, inequality, and poor governance remain significant barriers. As Cheung summed it up: “When one person is unhappy, that’s an individual issue. But when a population isn’t happy, that’s a structural problem—and it requires structural solutions.”

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PBS to Cover Ketamine-Based Nasal Spray for Treatment-Resistant Depression

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Updated May 1, 2025 | 11:00 AM IST

PBS To Cover Ketamine-Based Nasal Spray For Treatment-Resistant Depression

SummaryUnlike older medications that target serotonin – a “feel-good” chemical in the brain – the ketamine-based nasal spray works on a different neurotransmitter called glutamate. Read on to know more.

A new ketamine-like nasal spray will soon be available at a subsidised cost in Australia to help thousands living with treatment-resistant depression. The medication, called Spravato (esketamine), has been added to the Pharmaceutical Benefits Scheme (PBS), marking a significant shift in how the country approaches difficult-to-treat mental health conditions.

What Is Esketamine and How Does It Work?

Esketamine is chemically related to ketamine, a well-known anaesthetic that gained notoriety for recreational use in rave culture. However, in controlled medical settings, this compound is offering new hope for patients who have not responded to traditional antidepressants.

Unlike older medications that target serotonin – a “feel-good” chemical in the brain – esketamine works on a different neurotransmitter called glutamate. This chemical is believed to play a key role in restoring neural connections that influence mood regulation. The result is often a much faster response, with some patients feeling relief within hours instead of weeks or months.

Why It Matters: A Breakthrough After Decades

Esketamine is the first government-funded drug for major depression in over 30 years that works via a new mechanism. While most antidepressants developed since the 1980s have focused on serotonin or other monoamines, esketamine offers an alternative path by targeting glutamate and related brain circuits.

For those who have tried multiple medications without success, this drug represents a long-awaited option. Clinical trials in Australia show that nearly 50% of patients with treatment-resistant depression reported significant improvement after using esketamine.

Who Can Access It and What Will It Cost?

Starting Thursday, up to 30,000 Australians will be able to access Spravato through the PBS. Patients will pay $31.60 per dose, or $7.70 for pensioners and concession card holders. However, this does not include additional healthcare and administrative fees. Because of safety requirements, the drug must be administered under supervision at certified treatment centres, which will add to the overall cost.

Despite these extra expenses, making esketamine available through PBS is expected to reduce financial barriers and give more Australians access to potentially life-changing care.

Why Esketamine Stands Out

Since receiving approval from the US Food and Drug Administration (FDA) in 2019, esketamine has been used in hospitals and clinics across America, particularly in emergency care for severe depression. Unlike some drugs that sedate or numb emotions, esketamine is reported to alleviate core depressive symptoms — including intense feelings of hopelessness and suicidal thoughts — without dulling awareness.

Mental health experts say this rapid effect can be life-saving in critical cases where waiting for standard medications to take effect is not an option.

A Step Forward After Long Delays

The inclusion of Spravato in the PBS comes after a four-year wait and four separate funding submissions. The listing is seen as a major win for mental health advocates and patients who have pushed for broader access to advanced treatments.

While it may not work for everyone, esketamine opens a new chapter in treating depression — one focused on faster, more targeted relief for those who need it most.

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King Charles Opens Up About His Cancer Treatment, Calls Himself 'One Of The Statistics'

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Updated May 1, 2025 | 09:16 AM IST

King Charles Opens Up About His Cancer Treatment, Calls Himself 'One Of The Statistics'

SummaryKing Charles described himself as one of the "statistics" among the 390,000 who "sadly" receive a cancer diagnosis in the UK each year.

British King Charles recently reflected on his experience with cancer, saying it brought into "sharp focus the very best of humanity", while acknowledging that each new case is "a daunting and at times frightening experience" for those receiving a diagnosis and for their loved ones. The British monarch was diagnosed with an enlarged prostate gland in February last year, after which he has been getting regular treatments and receiving outpatient care. Despite the diagnosis at 77 years, he has remained positive about his treatment and has continued to perform some of his constitutional duties, such as paperwork and private meetings.

King Charles's Personal Message To People

In a personal written message, released to coincide with a Buckingham Palace reception celebrating organisations that help people with the disease, he described himself as one of the "statistics" among the 390,000 who "sadly" receive a cancer diagnosis in the UK each year. He said it "can also be an experience that brings into sharp focus the very best of humanity". Notably, he told the representatives of the cancer charity that they have the British royal family's "deepest admiration."

Dame Deborah James Is My Inspiration

King Charles hailed the late Dame Deborah James as an inspiration, quoting her final message and encouraging everyone to follow her example. "Find a life worth enjoying; take risks; love deeply; have no regrets; and always, always have rebellious hope." A royal source said there was no update on the king’s condition or treatment, but that it continued in a "very positive direction", reflected in his "very full" national and international diary. They described the words and subject of the message as "deeply personal" for the king.

While Buckingham Palace has not made an announcement clarifying the type of cancer the 76-year-old monarch has been battling but it is suspected that he might have prostate cancer. As described by UK NHS, prostate cancer is a type of cancer that occurs when malignant cells form in the prostate gland, which is a walnut-sized gland in the male reproductive system. Prostate cancer treatment guidelines have shifted their path a bit in recent years, with many men opting for active surveillance rather than immediate treatment for slow-growing tumours. However, about 50% of men on "watchful waiting" will require further treatment within 5 years because of the tumour progression. This is what triggered many researchers to aim and identify whether dietary modifications, specifically increasing omega-3 fatty acids, could prolong this surveillance period and slow down the tumour progression.

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