New Drugs Approvals – Asrar Qureshi’s Blog Post #929

New Drugs Approvals – Asrar Qureshi’s Blog Post #929

Dear Colleagues! This is Asrar Qureshi’s Blog Post #929 for Pharma Veterans. Pharma Veterans Blogs are published by Asrar Qureshi on its dedicated site https://pharmaveterans.com. Please email to aq.pharmaveterans@gmail.com for publishing your contributions here.

Credit: Mikhail Nilov

Credit: Pavel Danilyuk

Credit: Polina Tankilevitch

Periodically, I gather information and write a blog post about approval of new drugs in the USA, because it leads in the research, discovery, and commercialization of new, research products. Even if a pharmaceutical product is first registered in Europe, it is inevitably applied to USFDA for approval. USA also leads because it is the single largest market in the global pharma market. US accounts for about 47% of the world pharmaceutical market which is currently estimated to be about US$ 1.2 trillion. You can have an idea of the size. 

The list of new drugs/ newer uses of existing drugs is given below. 

DUVYZAT (givinostat) injection

Duvyzat is from Italfarmaco Group and is indicated for Duchenne Muscular Dystrophy – DMD. DMD is one of the most severe forms of inherited muscular dystrophies. It usually affects boys in early childhood who may live into their 20s and 30s only.

TRYVIO (aprocitentan) injection

Tryvio is an endothelin receptor antagonist for the combinations treatment of hypertension that is not adequately controlled with other drugs. ERAs are type of targeted therapy used to treat people with pulmonary hypertension These therapies may slow the progression of pulmonary hypertension and may even revers some of the damage to heart and lungs. The drug was applied by Idrosia Limited.

LENMELDY (atidar sasgene autotemcel) injection

Applied by Orchard Therapeutics, Lenmeldy sasgene is a gene therapy for the treatment of Metachromatic Leukodystrophy, a disease passed from parent to child through a broken gene. In MLD, the body cannot properly make an enzyme, which leads to loss of myelin, the special coating that protects body’s nerves.

REZDIFFRA (resmetirom) tablets

Rezdiffra is a thyroid hormone receptor-beta agonist indicated in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis – NASH, with moderate to advanced liver fibrosis. 

NASH is a serious form of fatty liver disease that causes the liver to swell and become damaged due to fat deposits in the liver. NASH may get worse and may lead to liver scarring, called cirrhosis, and even liver cancer. The damage is like the damage caused by heavy alcohol use.

The first line of management involves lifestyle modifications, sustained weight loss, and increased physical activity.  So far, the two best drug options, affirmed by the American Association for the Study of Liver Diseases, are vitamin E and pioglitazone. Rezdiffra offers a new mechanism for treating NASH.

Globally, 20-30% adults suffer from nonalcoholic fatty liver disease – NAFLD – of which NASH is an advanced stage. NASH market shall grow from $3.33 billion in 2023 to $4.54 billion in 2024. The approval of Rezdiffra to Madrigal Pharmaceuticals is therefore quite exciting.

TEVIMBRA (tislelizumab-jsgr) injection

BeiGene Ltd. received approval for Tevimbra, indicated for esophageal carcinoma. Tevimbra is a programmed death receptor-1 (PD-1) blocking antibody indicated for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma, after prior chemotherapy which did not include a PD-(L)1 inhibitor.

EXBLIFEP (cefepime and enmetazobactam) injection

Cefepime, a fourth generation, cephalosporin antibiotic has been around for several years. This is a new combination with a new beta lactamase inhibitor. Allecra Therapeutics received approval of Exblifep for the treatment of complicated urinary tract infections. 

ALGYLO (immune globulin intravenous, huma-stwk) liquid for intravenous injection

Algylo is a 10% immune globulin liquid for intravenous injection indicated for the treatment of primary immunodeficiency syndrome.

iDOSE TR (travoprost) intracameral implant

iDose TR is a long-duration prostaglandin indicated for the reduction of intraocular pressure in patients with open-angle glaucoma, or ocular hypertension. Presently, the patients have to put eye drops two or more times a day to keep the pressure under control. 

Discussion & Sum Up

The above is a selection from the very recent approvals from USFDA. I did not add many other drugs which include new mAbs and biosimilars of previous mAbs. 

While the world is advancing at a very rapid pace, we in Pakistan are regressing in every way. There is no research for drug development in any institution of the country. Our universities are dishing out PhDs whose research is not even worth the paper on which it is published. Intellectual dishonesty is at its worst at present. 

Our pharmaceutical industry is very excited to take over the entire pharma market, after driving out most of the multinational companies; long live patriotism. The few MNCs left are surviving by doing in Rome as the Romans do. The industry is neither geared nor interested in doing research. They are only churning out tablets, capsules, injections of the same medicines which everyone is doing. The name of the game is volume, not innovation; it is quantity, not quality. 

In this scenario, when we look at the development in the other world, even in neighboring India and Bangladesh, we can only feel ashamed. We should know that we shall not be able to get new products for our market due to our abysmal record of patent and contractual violations. 

It is a sad commentary that our patients shall remain deprived of advancements in the medical fields and that they will be forced to survive like this if they can. So far, government policies or government institutions such as DRAP – Drug Regulatory Authority Pakistan, are not doing any effort towards improvement.

Concluded.

Disclaimers: Pictures in these blogs are taken from free resources at Pexels, Pixabay, and Google. Credit is given where available. If a copyright claim is lodged, we shall remove the picture with appropriate regrets.

For most blogs, I research from several sources which are open to public. Their links are mentioned under references. There is no intention to infringe upon anyone’s copyrights. If, however, it happens unintentionally, I offer my sincere regrets.