FDA Approved: Turalio | First Therapy | TGCT Tumors | تمت الموافقة عليه من قِبل إدارة الأغذية والعقاقير | العلاج الأول | أورام

 

U.S. Food and Drug Administration granted approval to Turalio (pexidartinib) capsules for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery. Pexidartinib is indicated at 400mg taken orally twice daily on an empty stomach at least 1 hour before or 2 hours after a meal or snack, available in 200mg tablets.

TGCT is a rare tumor that affects the synovium (thin layer of tissue that covers the surfaces of the joint spaces) and tendon sheaths (layer of membrane that covers tendons, which are fibrous tissue that connect muscle to bone). 

The tumor is rarely malignant but causes the synovium and tendon sheaths to thicken and overgrow, causing damage to surrounding tissue.

The FDA granted this application Breakthrough Therapy designation and Priority Review designation. Turalio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Turalio to Daiichi Sankyo.

The prescribing information for Turalio includes a Boxed Warning to advise health care professionals and patients about the risk of serious and potentially fatal liver injury. Health care professionals should monitor liver tests prior to beginning treatment and at specified intervals during treatment. If liver tests become abnormal, Turalio may need to be withheld, the dose reduced, or permanently discontinued, depending on the severity of the liver injury. 

Turalio is available only through the Turalio Risk Evaluation and Mitigation Strategy (REMS)Program

 

 

  













 

Pharmaceutical Industry: Regulatory Affairs | India | دور الشؤون التنظيمية في صناعة الأدوية في الهند

The proposed reforms in the existing drug regulatory system, including allowing manufacturing and stockpiling of non-COVID vaccines while witnessing clinical trial. the Health Ministry's May 18, 2020, review announcement, saying it allowed manufacturing and stockpiling of COVID- 19 vaccine under clinical trial for marketing authorization for trade or distribution.

Because of this rule, it came possible for manufacturing and stockpiling the COVID- 19 vaccine during the clinical trial and they could make the vaccine available in such a short span of time to cover millions of lives.

By the end of 2020, SII( Vaccine Manufacturer) has formerly produced around 50 million tablets of the Oxford- AstraZeneca COVID- 19 vaccine" Covishield", indeed while it awaited the medicines Controller General of India'' s( DCGI) nod for exigency use of its vaccine in the country.

In view of the successful result of this provision for COVID- 19 vaccine, this provision should also be enforced fornon-COVID-19 vaccines, of authorization to use the remaining amounts of batches of COVID and non-COVID vaccines for marketable purpose which have been used in a clinical trial.

In this environment, the Health Ministry had issued draft rules dated April 12, 2018, to allow the remaining amounts of batches of vaccines which have been used in a clinical trial for marketable use after the entitlement of authorization in Form 46(now it's Form CT- 23) and manufacturing license in Form 28D. still, this draft rule has not been enforced till now. This draft rule should be enforced shortly to avoid destruction of life- saving vaccines.

Also perpetration of recommendations of inter-ministerial commission for reforming the medicine Regulatory Systems in India. The letter stated that on the directions of Prime Minister Narendra Modi, a high- powered inter-ministerial commission for reforming the medicine Regulatory Systems in India was constituted on May 11 last time under the chairmanship of also officer on special duty Rajesh Bhushan, who's presently the union health clerk. series of meetings of this commission was held starting May 2020.

Recommendations of this inter-ministerial commission should be enforced incontinently in line with the ease of doing business. Putting forward the following points with relation to necessary nonsupervisory reforms in the being Drug Regulatory system for your kind reference and intervention, which will take the vaccine assiduity of our country to new heights in the world.

The high minister's vision mentioning," It's a matter of great pride for all of us that because of our Prime Minister's vision about the nonsupervisory reforms, the vaccine assiduity of our country is growing veritably presto and under his global leadership, India has proved that as world leader in vaccine sector.

FDA: Accelerated Approval | Process | New Drugs | عملية الموافقة المعجلة لإدارة الغذاء والدواء على الأدوية الجديدة

 

The FDA has an accelerated approval mechanism for new drugs. Is accelerated FDA approval a good thing? Probably Yes. This mechanism of approval was put into place to help patients who suffer from certain serious diseases.

As the phrase suggests accelerated FDA approval involves a faster approval than for non-accelerated approvals. To get an accelerated approval sponsors must submit their application to the FDA. Before an application is accepted for approval, two major regulatory tests described below are applied. 

 

THE DRUG HAD TO TARGET SERIOUS CONDITIONS 

What is a serious condition? If you are a patient almost every disease would be a serious condition. The FDA has clarified this in a guidance document. 

This is a disease or condition which has significant impacts on a person's day-to-day ability to function or even die. 

Clinical judgment has to be made in determining whether a disease is serious or not. 

The judgment is based on the impact of the disease on factors such as the patients' survival and day-to-day functioning. In addition, an assessment is made on the likelihood that the disease will progress to a more severe condition if no treatment is provided. 

There are other three pathways for expedited approval of NDA which require establishment of serious condition. They include fast track, breakthrough therapy and priority review. 

All diseases that are life threatening qualify as serious illnesses but not necessarily for accelerated FDA approval as we will see later. Some will qualify for fast track, breakthrough therapy and priority review but not for accelerated approval.

 

THE DRUG HAD TO BE FOR AN UNMET MEDICAL NEED 

The term "unmet t Medical Need" is used to refer to a medical condition that does not have adequate treatment or diagnosis. It may be for treatment of cancer or a method for better and faster diagnosis of a serious illness. The application of the term "unmet medical need" could be quite broad.

The term "unmet medical need" could be used where there is no available appropriate treatment to the patients. Sometimes a new therapy may be available but unmet medical need may still exist such as when the new reduces the symptoms but not the serious outcome such as progressive disability or progression of the illness. 

 

WHAT THE SPONSOR SHOULD DO

Because many sponsors may want to use accelerated FDA approval, certain pre-qualification criteria have been put into place.

As the accelerated FDA approval application is based on surrogate endpoints. Sponsors should ordinarily discuss with the FDA of the intention to request for accelerated approval. 

The request would help the sponsor to choose the best surrogate endpoints during the clinical trials. Surrogate endpoints are endpoints other than clinical endpoints which take longer to determine for diseases that take long to show a clinical endpoint. 

For example, it might take long to show that a drug prolongs life. Prolongation of life is a clinical endpoint. Decrease in the blood in viral load such as in HIV would be a surrogate endpoint.

Surrogate endpoints include laboratory measurements and images among others. The use of clinical endpoints is however required in the confirmatory clinical trial reports.

By the time this application is being submitted the sponsor should be in a position to discuss the confirmatory clinical trials which should be in progress. 

Without evidence of these confirmatory trials the FDA may be reluctant to approve the requested accelerated FDA approval. The risk to patients is exposure to expensive drugs which may offer no clinical benefits. The sponsor must provide evidence for commitment to complete the pending confirmatory trials.

Does Canada have a similar program to accelerated FDA approval? The Canadian equivalence is called priority review. In this program the sponsor is encouraged to request for priority review status early. The sponsor will get a notice of compliance status with conditions after the application when the only data available is from surrogate end-points. 

The target review period is 200 days. Just as in USA, the sponsor has to submit confirmatory clinical trial data for full compliance without conditions.