Tag: FDA

Categories : Diseases, Syndromes and Conditions New Compounds and TreatmentsTags :

FDA Approves First Drug Designed to Treat Indolent Systemic Mastocytosis

On By ModernMedia
Photo by Rodion Kutsaiev on Unsplash

Systemic mastocytosis (SM) is a rare haematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients’ quality of life, and now the first medicine has been approved to specifically treat the most common form of the disease. On Monday 22 May, the US Food and Drug Administration (FDA) approved AYVAKIT® (avapritinib) to treat indolent systemic mastocytosis (ISM) in adults.

ISM represents the vast majority of SM cases, and AYVAKIT is now available for adults with ISM at the recommended dose of 25mg once daily. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. AYVAKIT has been FDA approved for the treatment of advanced SM since June 2021.

“After decades of caring for people with indolent systemic mastocytosis, I have seen firsthand its profound impact on patients’ underlying mast cell burden, symptoms, physical and mental health, and ability to work and participate in daily activities,” said investigator Cem Akin, MD, PhD, Professor of Medicine at the University of Michigan. “Despite the use of multiple supportive care treatments, a considerable number of patients with indolent systemic mastocytosis continue to experience a substantial disease burden. AYVAKIT advances the treatment of indolent systemic mastocytosis by targeting KIT D816V, the primary underlying cause of the disease, and establishes a new standard of care for a broad population of patients with this disorder. AYVAKIT delivered statistically significant and consistent clinical improvements in the PIONEER trial, and based on these practice-changing data, I feel a tremendous sense of hope for the future for all those affected by the disease.”

The approval of AYVAKIT in ISM is based on data from the double-blind, placebo-controlled PIONEER trial – the largest study ever conducted for this disease – in which patients received AYVAKIT 25mg once daily plus best supportive care (AYVAKIT) or placebo plus best supportive care (placebo). AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

AYVAKIT was well-tolerated with a favourable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity. The most common adverse reactions for AYVAKIT (≥10%) were eye oedema, dizziness, peripheral oedema and flushing. Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1% of patients.

Detailed results from the PIONEER trial, including open-label extension study data showing the clinical benefits of AYVAKIT through 48 weeks of treatment, were presented in February 2023 at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting.

Source: Blueprint Medicines

Categories : Ethics and Law Medical IndustryTags :

Did the FDA Break its Own Rules in Approving New Antibiotic?

On By ModernMedia
Photo by Rodion Kutsaiev on Unsplash

In the US, drug approval needs “substantial evidence” of effectiveness – but an investigation by The BMJ into the recent approval of the antibiotic Recarbrio from Merck suggests that these standards are being bypassed.

Recarbrio is a combination therapy made up of a new beta-lactamase inhibitor (relebactam) and a decades old Merck antibiotic (imipenem-cilastatin) to treat complicated infections. It costs $4000–$15 000 for a course, compared with a couple of hundred dollars for the generic version of Merck’s old antibiotic.

Peter Doshi, senior editor at The BMJ, describes how US Food and Drug Administration (FDA) scientists had serious doubts about its highly expensive Recarbrio but the agency approved it anyway.

Did the FDA break its own rules in approving this antibiotic, and what does this case tell us about problems within the agency, he asks? 

In its FDA application, Merck submitted results from two clinical trials comparing Recarbrio with imipenem in adults with complicated urinary tract infections and in patients with complex intra-abdominal infections.

But FDA reviewers noted that Merck had studied the wrong patient population to evaluate the added benefits of the new drug, and said the trial for urinary tract infections showed that Recarbrio was as much as 21% less effective than the older, cheaper imipenem.

The FDA concluded that “these studies are not considered adequate and well-controlled.” And of a third clinical study, the FDA called it a “very small,” “difficult to interpret” “descriptive trial with no pre-specified plans for hypothesis testing.”

Yet despite all three clinical studies not providing substantial evidence of effectiveness, FDA approved Recarbrio.

“Instead of basing its decision on the clinical trials in Merck’s application, FDA’s determination of Recarbrio’s efficacy was justified on past evidence that imipenem was effective, plus – to justify the new relebactam component – in vitro (lab) studies and animal models of infection rather than evidence from human trials as required by law,” writes Doshi.

Others are concerned that Recarbrio’s approval essentially amounts to a return to a way of regulating medicines that the FDA abandoned a half century ago prior to the agency’s “substantial evidence” standard.

Doshi explains that, under specific circumstances, the Director of the Center for Drug Evaluation and Research (CDER) can waive in whole or in part the FDA’s “adequate and well-controlled studies” approval criteria. But the FDA told The BMJ ”there was no center director memo in the file” for Recarbrio.

And when The BMJ contacted Janet Woodcock, CDER Director at the time, and now the FDA’s Principal Deputy Commissioner, she said she was not aware that clinical studies showed Recarbrio did not provide substantial evidence of effectiveness.

Woodcock was also unable to confirm that approvals of new drugs require at least one clinical study of the drug itself that demonstrates substantial evidence – evidence lacking in the case of Recarbrio.

A spokesperson for CDER told The BMJ that FDA “applied regulatory flexibility” in approving Recarbrio. 

It is unclear whether this regulatory flexibility enabled FDA to conclude Recarbrio had met the legal “substantial evidence” standard without “adequate and well-controlled investigations” of Recarbrio, says Doshi. FDA declined to answer the question, saying “We have no additional information to provide.”

The decline of science at the FDA has become unmanageable, argues David Ross, associate clinical professor of medicine at George Washington University, School of Medicine and Health Sciences, and former FDA medical reviewer, in a linked commentary.

He describes Recarbrio’s approval as “shocking” and says while much of the blame must go to the FDA’s reliance on industry paid user fees for around two-thirds of its annual drugs budget, “the corruption of the FDA’s scientific culture remains the primary culprit driving the deterioration of safety and effectiveness standards.”

To address this “dismal situation” he suggests tapering the FDA’s dependence on user fees and improving public access to the information received by the FDA, its reasoning, and its decisions.

“The Recarbrio approval is a sentinel event, warning of a return to an era when drug effectiveness was an afterthought,” argues Ross. “Although the FDA crowed about this approval, it would have been better advised to remember that “for a successful technology, reality must take precedence over public relations, for nature cannot be fooled,” he concludes.

Source: EurekAlert!

Categories : Pain ManagementTags :

New Migraine Prevention Drug Gets The Green Light from FDA

On By ModernMedia
Source: Usman Yousaf on Unsplash

Atogepant (Qulipta) has become the first oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) specifically developed for migraine prevention to win FDA approval, manufacturer AbbVie announced on Monday.

Following on after rimegepant, which is also indicated by the FDA for acute migraine treatment, atogepant became the second gepant approved for prevention of episodic migraine in adults.

The atogepant decision “reflects a broader shift in the treatment and management paradigm for the migraine community,” noted Peter Goadsby, MD, PhD, DSc, of the University of California Los Angeles and King’s College London.

“Qulipta provides a simple oral treatment option specifically developed to prevent migraine attacks and target CGRP, which is believed to be crucially involved in migraine in many patients,” said Dr Goadsby in a statement.
Atogepant has a high affinity at the CGRP receptor, and being a small-molecule drug it can be taken orally, unlike injectable anti-CGRP monoclonal antibodies approved for migraine prevention.
An oral CGRP-receptor antagonist is easier for patients, Goadsby noted when he presented data from atogepant’s pivotal phase IIb/III trial at the 2019 American Academy of Neurology annual meeting. “It could facilitate, with time, the greater use of this mechanism in primary care,” he told MedPage Today. “Primary care doctors will more easily use a medicine that’s relatively simple to use and well-tolerated, and that means more migraine patients can get treated.”

In the phase III ADVANCE trial, 873 participants were randomised to receive a once-daily dose of oral atogepant (10mg, 30mg, or 60mg) or placebo. After 12 weeks, average days with migraine per month dropped from baseline by 3.7 days with atogepant 10mg, 3.9 days with atogepant 30mg, 4.2 days with atogepant 60mg, and 2.5 days with placebo. The most common adverse events with atogepant were constipation and nausea, along with fatigue. 
Patients should notify their healthcare provider if they have kidney problems or are on dialysis, have liver problems, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed, AbbVie said.

Source: MedPage Today

Categories : COVIDTags :

Pfizer Vaccine Fully Approved in US

On By ModernMedia
Photo by Mat Napo on Unsplash

On Monday, the US Food and Drug administration approved the Pfizer/BioNTech COVID vaccine, the first vaccine against the novel coronavirus to receive full approval.

The vaccine, which will be marketed as Comirnaty, can be used for individuals ages 16 and older for COVID prevention. However, the vaccine is still under emergency use authorisation (EUA) for adolescents ages 12-15, the agency said.

FDA Acting Commissioner, Janet Woodcock, MD, said in a statement: “While this and other vaccines have met the FDA’s rigorous, scientific standards for emergency use authorisation, as the first FDA-approved COVID vaccine, the public can be very confident that this vaccine meets the high standards for safety, effectiveness, and manufacturing quality the FDA requires of an approved product.”

At a media briefing, Peter Marks, MD, PhD, the director for the Center for Biologics Evaluation and Research at FDA, detailed the meticulous process used for conducting a review of a biologics license application for full approval, including invidivual analysis of adverse events.

Since 11 December, 2020, the vaccine has been under an EUA for individuals ages 16 and older. Evidence for the full approval comes from expanded phase III trial safety and efficacy data released by the manufacturer this April. An analysis of 927 confirmed cases in the trial’s 44 000 participants found that 7 days after the second dose, Comirnaty had a 91.3% efficacy against symptomatic COVID through 6 months.

More than half of the participants had over 4 months of safety data, including 12 000 people who were followed up through 6 months. Injection site pain, fatigue, headache, muscle or joint pain, and fever were the most common adverse events. A slight increase in risk for myocarditis and pericarditis up to 7 days after the second dose was noted, particularly in males under 40 (peaking in ages 12-17), but symptoms resolved completely.

Trial data was collected before the Delta variant became the dominant strain in the US, Dr Marks noted, Israeli “real world” suggest the vaccine still retains effectiveness but wanes. This is something the agency will follow. 

Former FDA commissioner Dr. Mark McClellan, who now directs the Duke-Margolis Center for Health Policy, spoke to the media about the approval, saying surveys showed that it will help sway vaccine holdouts.

“I do think it will make a difference. Maybe not a large number of people running out and getting a vaccine today. At this point we’ve got a little bit over 70 percent of Americans who are eligible for the vaccine have gotten at least one dose. That’s about 87 million Americans who are eligible who haven’t. Out of those, according to some recent surveys, about 30 percent say the full approval of the Pfizer vaccine would make a difference in their decisions.”

Source: MedPage Today

Categories : New Compounds and TreatmentsTags :

New Lupus Treatment Gets the Nod from FDA

On By ModernMedia
Source: Unsplash

AstraZeneca announced that its type 1 interferon receptor antagonist anifrolumab (Saphnelo) has received approval from the US Food and Drug Administration for the treatment of moderate-to-severe systemic lupus erythematosus alongside standard therapy.

“This is wonderful, exciting news, and is great for the lupus community — patients, family members, and clinicians who treat patients,” said Richard Furie, MD, chief of rheumatology at Northwell Health in Great Neck, New York, in an interview.

Only belimumab (Benlysta) in 2011 and voclosporin (Lupkynis) for lupus nephritis a few months ago had been approved in the past decades. “And that represents 25 years of trying,” Dr Furie said.

Significant benefits were reported in 2016 in a phase IIb trial known as MUSE. In that trial, 62.6% of patients receiving 300 mg intravenous anifrolumab every 4 weeks had an SLE Responder Index score of 4 (SRI-4) plus a reduction in the steroid dose to less than 10 mg/day compared with only 17.6% of patients in the placebo group, which was a significant difference — the best lupus trial data so far, according to Dr Furie.

Two pivotal phase III trials, TULIP-1 and TULIP-2, followed, with conflicting results.

In TULIP-1, the primary endpoint of Systemic Lupus Erythematosus Responder Index (SRI-4) was not met. After a year, an SRI-4 response was seen in 36% of patients receiving anifrolumab and in 40% on placebo. Some secondary endpoints suggested benefits, including the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA).

In TULIP-2, patients were randomised 300 mg intravenous anifrolumab or placebo every 4 weeks for 48 weeks, with a BICLA response as the primary endpoint. After a year, 47.8% of patients in the anifrolumab group achieved a BICLA response compared with 31.5% of placebo patients.

“We were all shocked when TULIP-1 failed,” said Dr Furie, who is also a leading member of the Lupus Research Alliance’s Lupus Clinical Investigators Network. “But it didn’t really fail — it depends on how you define failure. It did not reach the primary endpoint, but on the other composite, BICLA, it was successful, as well as on a lot of the key secondary endpoints. The totality of the data, I think, is the key phrase,” he said.

“I think the two studies were more similar than dissimilar. You have to have an appreciation of how difficult it is doing clinical trials in lupus. For every one trial that has been successful, there have probably been 10 that were unsuccessful,” he said.

This was not the first time discordant results had been seen in lupus trials. “We also saw discordance between the BICLA and SRI in the ustekinumab phase II trial,” he noted.

Source: MedPage Today

Categories : Medical IndustryTags :

Over 100 Fast-tracked Drugs Not Confirmed Effective

On By ModernMedia

Of 253 drugs approved via the FDA’s accelerated approval pathway, clinical effectiveness has been confirmed in 112, according to a new investigation by The BMJ.

Clinical reporter Elisabeth Mahase found that, as of the end of last year, 24 of those 112 drugs have been on the market for more than 5 years, and some have been on the market for more than 2 decades — often with a high price tag, according to.

Though the accelerated approval pathway allows drugs onto the market before efficacy has been established, the manufacturer has to perform confirmatory trials or else the approval will be rescinded.

However, Mahase noted that only 16 drugs authorised through the accelerated approval pathway have been withdrawn since its creation in 1992 . Most of those were shown to lack efficacy, but in some cases, confirmatory trials were simply never done. Celecoxib (Celebrex), for example, was given accelerated approval in 1999 for the treatment of familial adenomatous polyposis, a genetic disorder that carries a high risk of bowel cancer if untreated, remained on the market for about 12 years before the FDA asked Pfizer to voluntarily withdraw it for this specific indication because efficacy trials were never completed.

The BMJ asked the manufacturers of 24 drugs that have been on the market for more than 5 years if they had conducted phase IV trials. Six drugs had been withdrawn, approved, or postponed. Of the remaining 18 drugs, relevant trial information was provided for a third. Four manufacturers of those six drugs were recruiting participants, and two reported talking to the FDA about final trial design. Eleven companies representing 12 drugs did not respond.

FDA response

“We are committed to ensuring the integrity of the accelerated approval program, which is designed to bring safe and effective drugs to patients with unmet medical needs as quickly as possible,” an FDA spokesperson said in a statement provided to MedPage Today. “The program allows the FDA to approve a drug or biologic product intended to treat a serious or life-threatening condition based on an outcome that can be measured earlier than survival that demonstrates a meaningful advantage over available therapies.”

The FDA could choose to initiate proceedings to withdraw a drug’s approval should post-marketing trials show no benefit or not be performed in time, added the spokesperson.

“Because the FDA continues to use this pathway to accelerate access to drugs for serious and life-threatening diseases for which there is an unmet medical need, at any point in time there will be drugs that are not converted because the confirmatory trials are ongoing,” the spokesperson said.

“Despite the pathway’s good intentions to accelerate ‘the availability of drugs that treat serious diseases,’ experts are concerned that it is now being exploited, to the detriment of patients — who may be given a drug that offers little benefit and possible harm — and of taxpayers,” she continued.

Fixing the accelerated approval pathway

Concerns about the accelerated approval pathway include a lack of threats from the FDA to withdraw a drug should confirmatory trials not be done, the agency’s use of indirect (or surrogate) measures of clinical benefit in some cases, and the potential for drug manufacturers to take advantage of the pathway when it comes to actual measures of safety and effectiveness, Mahase wrote.

Nevertheless, experts still agree that the accelerated approval pathway is of benefit, she noted. Suggested changes to the pathway include planning or starting confirmatory trials as part of the approval as well as closer examination of surrogate measures.

A recent example is Biogen’s controversial Alzheimer’s disease treatment aducanumab (Aduhelm) received FDA approval via the process last month, which was based on the surrogate endpoint of reduction of amyloid-beta plaque in the brain.

The drug has attracted criticism since its recent fast-track approval, with critics pointing out that the drug has not been proven effective and its $56 000 annual price is unreasonable.

Source: MedPage Today

Journal information: Mahase E “FDA allows drugs without proven clinical benefit to languish for years on accelerated pathway” BMJ 2021; DOI: 10.1136/bmj.n1898.

Categories : Hospitals Implants and ProsthesesTags :

Deaths From Medical Devices Are Underreported in the US

On By ModernMedia
Photo by Vidal Balielo Jr. from Pexels
Photo by Vidal Balielo Jr. from Pexels

Researchers have found that a number of deaths related to medical device adverse events were improperly categorised in the FDA’s Manufacturer and User Facility Device Experience (MAUDE) database, according to a new study.

Flagging terms commonly associated with death, the study investigators used a natural language processing algorithm to identify 290 141 reports where serious injury or death was reported; 52.1% of these events were reported as deaths, and 47.9% were classified as either malfunction, injury, or missing (report was uncategorised), reported Christina Lalani, MD, of the University of California San Francisco, and colleagues, in JAMA Internal Medicine.

Overall, 23% of reports with a death were not placed in the death category, amounting to some 31 552 reports filed from December 31, 1991, to April 30, 2020.

Whether to classify the event as a malfunction, injury, death, or ‘other’ is up to the physician or manufacturer. According to the FDA, the reporter is required to categorise an adverse event as an official death if the cause of death is unknown, or if the device “may have caused or contributed to a death.”

The three most common product codes among the adverse event reports were for ventricular assist bypass devices (38 708 reports), dialysate concentrate for haemodialysis (25 261 reports), and transcervical contraceptive tubal occlusion devices (14 387 reports).

The natural language processing algorithm scanned through reports, identifying terms such as “patient died,” “patient expired,” “could not be resuscitated,” and “time of death.” Of the 70 terms that were associated with a death, 62 (88.6%) were found among miscategorised adverse event reports involving a patient death. And, out of all 62, there were 17 terms that had an estimated percentage of 100%, meaning that “every time that term was used, the patient had died, even though the reporter had not classified the report as death,” the team wrote.

Only 18 terms had sample sizes large enough for researchers to calculate confidence intervals; among them, the words “death” or “deaths” were linked to 12% of adverse event reports in which a patient died, but were classified as malfunction, other, or missing — the highest rate of all the analysed terms.

The researchers acknowledged a major limitation in that only reports with at least one death-associated term were included, in contrast to all the reports from the MAUDE database. Improperly categorised deaths likely contribute to an underestimate.

“The classification chosen by the reporter is vital, as the FDA must review all adverse events reported as deaths, which is not the case for other reporting categories,” the authors wrote. Improving the reports’ accuracy is crucial, since patient death frequency can prompt the FDA to pursue investigations into the device’s safety, they added.

The researchers pointed out an inherent conflict of interest as 95.9% of the reports evaluated in the study were submitted by manufacturers.

“It may not be in their interest to facilitate identification of serious problems with their own devices in a timely manner,” they wrote. “There have been multiple instances of delays by manufacturers in reporting serious malfunctions and deaths that were associated with medical devices, as well as complete failures to report.”

Therefore, it’s likely that a significant number of patients have been unknowingly treated with devices that were later revealed to be dangerous, Dr Lalani and colleagues noted. For example, they referenced the reporting failures that occurred from 2002 to 2013, when 32 000 women reported adverse events associated with the permanent birth control device Essure while the FDA only received 1023 adverse event reports from the manufacturer.

They concluded that patients and care providers should submit reports directly to the FDA as well as or instead of the manufacturer.

Source: MedPage Today

Journal information: Lalani C, et al “Reporting of death in US Food and Drug Administration medical device adverse event reports in categories other than death” JAMA Intern Med 2021; DOI: 10.1001/jamainternmed.2021.3942.

Categories : Neurodegenerative Diseases New Compounds and TreatmentsTags :

Probe over Controversial Alzheimer’s Drug’s Approval

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Amyloid plaques and neurons. Source: NIAH

The interim commissioner of the US Food and Drug Administration, Janet Woodcock, MD, last week requested the country’s Office of Inspector General to perform an independent investigation into the regulator’s decision to approve Biogen’s controversial Alzheimer’s drug Aduhelm.

Dr Woodcock noted in her letter that there “continues to be concerns raised” regarding the contact between FDA officials and Biogen ahead of the agency’s decision, “including some that may have occurred outside of the formal correspondence process.”

Dr Woodcock’s request comes after a bombshell report from Stat, which found that Biogen executives met with FDA officials, specifically Billy Dunn, MD, director of the FDA’s neuroscience unit, as early as 2019 to discuss a regulatory pathway for Aduhelm. The meetings took place even when it seemed there was no progress for the drug.

Earlier this week, a US House Representative, charged Biogen with “undue influence” over the FDA’s review process. Less than two weeks earlier, the House Committee on Oversight and Reform said it would conduct its own probe into the approval along with Biogen’s pricing strategies.

In the letter from Friday, Dr Woodcock said the agency would fully cooperate with the potential investigation to determine whether any of its interactions with Biogen were inconsistent with FDA policies and procedures.

“Given the ongoing interest and questions, today I requested that @OIGatHHS conduct an independent review and assessment of interactions between representatives of Biogen and FDA during the process that led to the approval of Aduhelm,” tweeted Dr Woodcock.

However, she maintained that she has “tremendous confidence” in the leadership at the FDA’s Center for Drug Evaluation and Research, which was involved in the review of Aduhelm.

“We believe this review and assessment will help ensure continued confidence in the integrity of FDA’s regulatory processes and decision-making,” Woodcock said in a tweet.

A spokesperson from Biogen told Fierce Pharma that the company would “of course” cooperate with “any inquiry in connection with a possible review of the regulatory process.”

The commissioner’s request is only the latest event in a bizarre and twisted story since the FDA’s Aduhelm approval just one month prior.

Facing fierce criticism of its wide-labelled approval, the FDA made the surprising move to narrow Aduhelm’s label last week Thursday, restricting the recommendation to just those with milder Alzheimer’s.

This comes after Biogen’s drug was essentially allowed access to the nation’s some 6 million Alzheimer’s patients. That decision was met with almost immediate pushback, as it was pointed out that the drug could overwhelm the payer budgets of most Alzheimer’s patients.

Source: Fierce Pharma

Categories : PaediatricsTags :

New ADHD Drug Gets FDA Approval for Children

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US regulators have approved the first new ADHD drug for children in over a decade.

The Food and Drug Administration last week approved  viloxazine (Qelbree) for the treatment of attention deficit hyperactivity disorder in children ages 6 to 17. It was developed by  Supernus Pharmaceuticals. The drug’s price was undisclosed but is likely to be higher than the generic ADHD pills.

In Europe, viloxazine was sold as an antidepressant for several decades, but never received FDA approval. It was discontinued nearly two decades ago, due to competition from popular pills like Zoloft and Prozac.

ADHD affects about 6 million American children and adolescents. For many, problems include trouble paying attention and completing tasks, fidgeting and impulsiveness.  

Earlier ADHD treatments like Ritalin, nearly all of which contain the stimulants amphetamine or methylphenidate, which create the potential for abuse. Viloxazine however is not a stimulant or a controlled substance. It carries a warning of potential for suicidal thoughts and behaviour, which occurred in fewer than 1% of volunteers in studies of the drug.

Qelbree could be an option for children with substance use disorders, who do not cope well with stimulant side effects or who need more therapy, said Dr David W. Goodman, director of Suburban Psychiatric Associates near Baltimore and an assistant professor of psychiatry at Johns Hopkins School of Medicine.

Goodman said that long-acting stimulants prescribed to ADHD patients currently are harder to abuse to get a high than the older fast-acting versions.

In a late-stage study, 477 children ages 6 to 11 took viloxazine for six weeks. Compared to placebo, Inattention and hyperactivity symptoms were reduced by about 50%. Symptom reduction was seen within a week in some participants. Its common side effects include sleepiness, lethargy, decreased appetite and headache.

Supernus is in late-stage testing for adults with ADHD, who represent a small but growing market as adult treatment of the condition expands.

Source: Medical Xpress

Categories : COVID VaccinesTags :

US Rollout of Johnson & Johnson Vaccine As It Gets FDA Approval

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Johnson & Johnson’s single shot COVID vaccine is set to roll out in the US after its approval, but concerns linger as to the public’s perception of its relative effectiveness.

The vaccine received an emergency use authorisation (EUA) on Saturday from the FDA, and received approval from the CDC the following day. On Sunday night White House officials stated that distribution of 3.9 million doses of the J&J vaccine would begin immediately, with J&J expecting to deliver some 16 million more doses by the end of March. These vaccines will be allocated proportionally, as per the procedure for Pfizer/BioNTech and Moderna vaccines.

At a Saturday media briefing, acting FDA Commissioner Janet Woodcock, MD, reiterated issues raised by the FDA advisory committee, that the J&J product’s lower efficacy number (70% vs 95%) may cause people to think it is less effective than the alternatives. She said that wasn’t necessarily so, urging Americans to “take the vaccine they are able to access.”

“All these vaccines meet our standards for effectiveness. They were not studied in head-to-head trials, so [they’re] difficult to compare … due to differences in development programs,” she said. (Preventing moderate-to-severe COVID illness was the J&J endpoint, whereas in the Pfizer and Moderna studies the endpoint was all symptomatic COVID.)

“We need to be clear on our messaging regarding comparisons with other vaccines,” said Jason Goldman, MD, of the American College of Physicians. “As a primary care physician, many of us are eager to vaccinate” patients and this vaccine will be “helpful in achieving that goal.”

Macaya Douoguih, MD, of J&J’s Janssen unit where the vaccine was developed, talked about the potential advantages of a one-dose vaccine, referencing the company experience with the Ebola vaccine

“For an outbreak setting, a single dose has a tremendous advantage in terms of being able to rapidly roll out mass vaccination” without the complexity of following up for a second dose, she said.

Dr Douoguih addressed the company’s planned two-dose study, saying that while a two-dose regimen might be “more immunogenic and lead to durable efficacy,” she thought there was room for both options. The two-dose option would be preferable in an ‘everyday’ COVID setting. The company was trying to enroll 16-17 year olds for additional data in a study starting next week, Dr Douoguih said.

The CDC researchers discussed preliminary data on asymptomatic infection, which assessed seroconversion between days 29 and 71. Those data showed vaccine efficacy against seroconversion was 74% (95% CI 48%-87%), but the CDC urged caution as the data was only preliminary.

“Our level of confidence in asymptomatic infection is tempered by low numbers and that is important for us to remember,” said Advisory Committee on Immunization Practices committee member Sarah Long, MD, of Drexel University College of Medicine in Philadelphia. “I appreciate the workgroup concluding the confidence is not that high.”

Source: MedPage Today