New Gene Therapy Products – Part 2 – Asrar Qureshi’s Blog Post #747

New Gene Therapy Products – Part 2 – Asrar Qureshi’s Blog Post #747

Dear Colleagues!  This is Asrar Qureshi’s Blog Post #747 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 asrar@asrarqureshi.com for publishing your contributions here.

Photo Credit: Gilmer Diaz Estela

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

We continue to look at some more drugs.

CARVYKTI (ciltacabtagene autoleucel) – has been launched by Janssen Biotech, Inc. it is approved for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti—CD38 monoclonal antibody. CARVYKTI price ranges from $465,000 to $489,000 for one dose. 

IMLYGIC (talimogene laherparepvec) – has been launched by Amgen Inc. It is indicated for the local treatment of unresectable cutaneous, subcutaneous, and nodal lesions in patients with melanoma recurrent after initial surgery. It is a multidose treatment which will cost around $65,000 for a course of therapy.

KYMRIAH (tisagenleclucel) – is a Novartis product. It is a CD19-directed, genetically modified T-cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma after two or more lines of therapy. It has been priced at $475,000 for one treatment course.

LUXTURNA (voretigene neparvovec-rzyl) – is a product from Spark Therapeutics, Inc. It is indicated for the treatment of patients with biallelic RPE65 mutation-associated retinal dystrophy. The cost of one-time treatment is $850,000. The drug can restore functional vision in patients with IRD – Inherited Retinal Disease.

PROVENGE (sipuleucel-T) – is from Dendreon Corporation. The therapy is indicated for the treatment of asymptomatic, or minimally symptomatic metastatic castrate resistant (hormone refractory) prostate cancer. The multicycle treatment will cost around $93,000.

SKYSONA (elivaldogene autotemcel) – is a prized product from Blurbird Bio. Inc. The therapy is indicated to slow down the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). At $3 million, it is the most expensive drug launched ever, even more expensive than the $2.8 million price tag of Bluebird’s other product ZYNTEGLO.

TECARTUS (brexucabtagene autoleucel) – is from Kite Pharma Inc. It is indicated for adult patients with relapsed or refractory mantle cell lymphoma (MCL). New indication – adult patients with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia. The one-infusion treatment cost is $373,000.

YESCARTA (axicabtagene ciloleucel) – is also from Kite Pharma Inc. The therapy is indicated for the treatment of adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy, or that relapses within 12 months of first-line chemoimmunotherapy. Axicabtagen ciloleucel is not indicated for the treatment of patients with primary central nervous system lymphoma. YESCARTA has the same price as that of TECARTUS at $373,000 for one treatment.

In addition, several products based on human cord blood taken from placenta of newly born children have also been registered by USFDA. These are for use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. Human cord blood of their children can be stored by the parents in a private blood bank who will charge a fee for keeping it, or they can donate it to a public bank for use by anyone else in need. The cord blood selling price is from $1600 to $1900 per pack. 

Other Conclusions

Few other things can be concluded from the discussion about innovative therapies.

• Medical science has made amazing progress. Finally, patient-specific treatments have become available along with targeted therapies. It would make treatment much more successful. Problem however is that these therapies have extremely serious side effects which cannot be avoided. And the whole list of adverse effects is not yet known. 

• Pricing has no relation to cost of production, or the cost of development. The rationale for pricing is the value it is expected to deliver, which it may or may not. This is a new philosophy never practiced before. Previously, drug prices were determined on the basis of manufacturing cost, plus margin to cover development cost, and of course a decent profit.

• Medical treatment in countries like ours and the developed world is progressing along two different trajectories. The diseases are not too different, but the therapies are becoming different. From conventional drugs which we use in Pakistan to the gene therapies being introduced rapidly, the outcomes are different. 

• The MNCs have found a novel way of fighting with generic companies; they are making mAbs and Nibs and cell-based therapies, and gene therapies which generic manufacturer shall find extremely hard to replicate. The supremacy and hegemony shall continue.

• Control over new drugs through very high pricing and limited production shall add another weapon to control the rest of the world. We have seen a trailer during COVID19 when the poor nations were begging for vaccine from Pfizer and J&J and Moderna. If China and Russia had not come to the rescue of these nations, they would not get the vaccine. Many reports surfaced that surplus quantities of vaccines were thrown away but were not given even at cost.

This is where the world is going. Progress is really commendable, but progress is not being used for the benefit of the people; it is being employed to gain more control, and huge profits.

Concluded.

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