Dear Colleagues!  This is Asrar Qureshi’s Blog Post #746 for Pharma Veterans. Pharma Veterans welcome sharing of knowledge and wisdom by Veterans for the benefit of Community at large. Pharma Veterans Blog is published by Asrar Qureshi on WordPress, the top blog site. Please email to for publishing your contributions here.

Gene therapy is among the newest modalities of treatment. It is still an experimental treatment that works by introducing genetic material into a person’s cell to fight or prevent disease. Gene Therapy is presently being tested for several diseases, such as autoimmune disorders, hemophilia, thalassemia, Parkinson’s disease, cancer, diabetes, heart disease, and so on. In this treatment, a gene can be delivered to a cell using a carrier called ‘vector’. Mostly, viruses are used as vectors due to their innate ability to penetrate the human cells. Of course, the disease-causing genes of viruses are taken out before using these as vector. Gene therapy is being tried for purposes other than fighting diseases as well.

Gene therapy drugs are to be given as a single dose, only once in life.

In a previous post, I had discussed the approval and availability of Novartis’s gene therapy product ZOLGENSMA, the first drug approved by USFDA in 2019 for Spinal Muscular Atrophy. It made headlines for two reasons; being the first USFDA approved gene therapy for this genetic disorder and being the most expensive drug at $2.1million per dose. Novartis reported two deaths in children after receiving ZOLGENSMA. The deaths occurred due to acute liver failure in Russia and Kazakhstan. EMA – European Medicine Agency is considering sending information to healthcare professionals containing guidelines and recommendations while administering the treatment.

Gene therapy has been moving forward, and new drugs have been approved for various indications with novel treatments. I shall review these in couple of blog posts.

USFDA approved Bluebird Bio’s gene therapy for patients with a rare blood disorder Beta Thalassemia, which requires regular blood transfusions. The drug has been priced at $2.8million per dose. The drug has been branded ZYNTEGLO. In their price calculation, the company has looked at the cost of blood transfusions over a lifetime which comes to $6.4million in the US, hence this price tag.

Thalassemia is not uncommon in Pakistan. Among other reasons, interfamily marriages, or cousin marriages over generations have increased the incidence greatly. Thalassemia is a major health hazard because the children suffer from many complications. Thalassemia causes reduction in hemoglobin and therefore the oxygen supply to vital organs, and overall health is compromised. These children remain weak and may not do well at school. To compensate for less hemoglobin, blood is transfused regularly, which leads to iron overload and its complications. Most Thalassemia patients die early due to poor health and complications.

Pakistan certainly needs this treatment, but the cost is so prohibitive, it is impossible to buy here. A single treatment of one dose will cost over 67 crore rupees. This is the economic inequality which we do not realize. Our patients are greatly supported by philanthropic organizations that run Thalassemia centers. They treat the patients at no cost, or highly subsidized cost. Fatimid and Sundas Foundation are well-known, but actually there are over 100 centers offering blood transfusions. Government is also running some centers. Without these free centers, most of our patients would die quite early. Life goes on.

The other gene therapy approved recently is HEMGENIX, treatment for hemophilia type B, caused by absence of clotting factor IX. CSL Behring has developed this drug and currently it is the most expensive drug in the world, with a price tag of $3.5million, which converts to 84 crore rupees per treatment, another impossible treatment. Type B is seen in about 15% of hemophilia patients; type A caused by the absence of factor VIII is more common, but no treatment is available yet. Fortunately, Hemophilia is relatively uncommon in Pakistan

Celgene Corporation has received approval for their gene therapy drug, ABECMA – idecabtagene vicleucel. The therapy is aimed at the treatment of patients with relapsed or refractory Multiple Myeloma after four or more prior lines of therapy including an immunomodulatory agent, a protease inhibitor, and anti-CD38 monoclonal antibody. The cost of one dose is listed at $419,500 but may cost up to half a million US dollars. Multiple myeloma is a type of bone marrow cancer. It is called ‘multiple myeloma’ as the cancer often affects several areas of body, such as spine, skull, pelvis, and ribs.

Ferring Pharmaceuticals has received approval for their gene therapy product, ADSTILADRIN – nadofaragene firadenoved-vncg. The therapy is for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. The approval came on 16 December 2022. Ferring Pharma said it would make the product widely available. The price is not finalized yet, but it would be around $200,000.

Juno Therapeutics Inc. has received approval for their gene therapy BREYANZI – lisocabtagene maraleucel – indicated for treatment of adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma, who have relapsed or refractory disease after two or more lines of systemic therapy. Lymphomas refer to types of cancer that begin in the lymphatic system and are the sixth most common form of cancer. The cost of BREYANZI is around $410,000.

We shall continue to explore more gene therapies in the next post. So far, the summary is that gene therapy is moving at a fast pace. They do offer a new hope for patients, but they carry a long list of very serious side effects, and a huge price tag.

 To be Concluded……

Disclaimer: Most pictures in these blogs are taken from Google Images and Pexels. Credit is given where known; some do not show copyright ownership. However, if a claim is lodged at any stage, we shall either mention the ownership clearly, or remove the picture with suitable regrets.

1 comment

  1. Very informative blog,a sincere effort for those who want to be well informed about profession.

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