Dear Colleagues!  This is Asrar Qureshi’s Blog Post #837 for Pharma Veterans. Pharma Veterans aims to share knowledge and wisdom from 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.

Photo Credit: Pavel Danilyuk

Periodically, I bring to you the update on new registrations by the USFDA. USFDA approval is most relevant because most research is happening in the US, and USFDA is mostly the first regulatory authority to approve new drugs, line extensions, new combinations, and expansion of indications. Some drugs are first applied to EMA – European Medicines Agency, but these are relatively few.

A look at new approvals is not just academic; it shows where the research is going, and where most investment is being done. It also reflects the incidence of diseases, unmet needs, and innovative treatment options. So, let us have a look at major, new approvals. I shall skip some products and much of the technical details to keep this post simple to comprehend.

New Approvals

ELREXFIO – elranatamab-bcmm Injection – accelerated approval granted to Pfizer on August 14, 2023

Elrexfio is a monoclonal antibody drug which is a B-cell maturation antigen for the treatment of patients with relapsed or refractory Multiple Myeloma.

HEPZATO – melphalan lyophilized powder for injection – approval granted to Delcath Ssytems Inc. on August 14, 2023

Melphalan was already available; the new approval is for a Hepatic Delivery System Kit for the treatment of adult patients with unresectable hepatic-dominant metastatic Uveal Melanoma.

AKEEGA – abiraterone acetate and niraparib Tablets – approval granted to Janssen Pharmaceuticals on August 11, 2023

Abiraterone had been available already; the combination is innovative. It has been approved for prostate cancer in adult patients, which is deleterious or suspected deleterious, BRCA-mutated, metastatic, and castration-resistant (mCRPC).

TALVEY – talquetamab-tgvs Injection – approval granted to Janssen Pharmaceuticals on August 9, 2023

Talvey is a monoclonal antibody drug, a first-in-class, bispecific therapy for the treatment of patients with heavily pretreated Multiple Myeloma.

ZURZUVAE capsules – zuranolone – approval granted to Sage Therapeutics on August 4, 2023

Zurzuvae is the first oral treatment for post-partum depression. Its approval made headlines on BBC and other channels. Post-partum depression is a common ailment in which the new mother feels low and depressed within 6 weeks after childbirth, rather than feeling happy and elated. It is not about having a baby boy or baby girl. The incidence of PPD is reported to be from 0.5% to 60% globally, and from 3.5% to 63.3% in Asian countries; Malaysia has the lowest and Pakistan has the highest rate at 28-63%. This means that this drug is urgently needed in Pakistan also.

RiVive – naloxone hydrochloride Nasal Spray – approval granted to Harm Reduction Therapeutics on July 28, 2023

Naloxone is an old drug which is opioid antagonist; it is given as emergency treatment in cases of Opioid Overdose. It is available in Pakistan also; the difference is that in the US it is available over-the-counter (OTC). The innovation is the nasal spray delivery system. Opioid overdose patients are not conscious to take oral drugs, but a nasal spray can still be given at home.

BALFAXAR – prothrombin complex concentrate, human-lans lyophilized powder for Injection – approval granted to Octapharma on July 21, 2023

Balfaxar is another very important approval. The drug is indicated for reversal of warfarin effect in urgent surgery and invasive procedures. Many patients are given warfarin, an old drug, as a blood thinning agent, but it increases the clotting time. Such patients may bleed excessively if surgery is done without stopping warfarin for several days prior to surgery. Balfaxar shall reverse the effect of warfarin so that emergency surgery or invasive procedures may be performed.

VANFLYTA – quizartinib Tablets – approval granted to Daiichi Sankyo Inc. on July 20, 2023

Vanflyta is a tyrosine kinase inhibitor indicated for the treatment of patients with newly diagnosed FLT3-ITD positive Acute Myeloid Leukemia.

BEYFORTUS – nirsevimab-alip Injection – approval granted to Astra Zeneca on July 17, 2023

The monoclonal antibody drug is indicated for the prevention of Respiratory Syncytial Virus lower respiratory disease in infants. RSV infection rate is highest in infants between 2-8 months and an estimated 4-5 million infants acquire RSV every year. RSV can deteriorate quickly if not treated immediately; of course, prevention is better.

OPILL – norgestrel Tablets – approval granted to Perrigo company on July 13, 2023

 Opill was another drug that made headlines in the US after the right for voluntary abortion at up to 12 weeks was revoked by the Supreme Court. It is a daily contraceptive, over-the-counter drug.

LANTIDRA – donislecel-jujn Cellular suspension for Infusion – approval granted to CellTrans Inc. on June 28, 2023

Lantidra is an allogeneic pancreatic islet (islets of Langerhans which produce insulin) cellular therapy for the treatment of type 1 diabetes mellitus in adults whose symptoms are not well controlled.

Two gene therapies have also been approved: one for the treatment of adults with severe hemophilia A; one for the treatment of ambulatory pediatric patients aged 4 though 5 years with Duchenne Muscular Dystrophy.

You would notice that cancer remains in focus for drug development, and gene therapies list is growing. However, few other breakthrough drugs have also been approved. Drug research and development is quite costly now, with cost estimates from discovery to approval going above one billion US$. To contain the costs, smaller agile research-only companies have been established whose primary job is to discover new drugs. These are then taken up by the Big Pharma for development and commercialization.

Pakistan has zero drug research for new drugs discovery; here, only formulation development is done. We are and we shall always be dependent on the developed countries for new drugs. The availability of new drugs to countries like ours will never be certain due to several factors, such as priorities, prices, patents etc.


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.


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