In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases. Rare diseases are those affecting fewer than 200,000 Americans.…
In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
According to a story in the WSJ, the FDA has approved a new therapy for multiple myeloma patients. The therapy is called Carvykti and it is now available as a…
According to a story from PR Newswire, Myeloma Action Month takes place every year during the month of March. This year, the International Myeloma Foundation (IMF) is organizing around the…
In Part 1 of our interview with Lorrie and Stewart Altman, we discussed the journey to the Late-onset Tay-Sachs disease (LOTS) diagnosis, how Lorrie and Stewart's outlook shaped their lives,…
On February 16, 2022, Healio shared that CT103A, a therapy for adult patients with relapsed or refractory (R/R) multiple myeloma (MM), had received Orphan Drug designation from the United States…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
According to a press release from the pharmaceutical company Bristol Myers Squibb, the company's CAR T-Cell therapy idecabtagene vicleucel (marketed as Abecma) has been approved by the Japanese Ministry of…
Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions. For the purpose of the designation, “rare” is considered any condition…
In a recent news release, biopharmaceutical companies AbbVie, Inc. and SpringWorks Therapeutics, Inc. ("SpringWorks") shared that the two had entered into a joint clinical trial collaboration agreement. Through this collaboration,…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, "rare"…
Since the onset of COVID-19, there have been approximately 262M cases, and 5.2M deaths, on a worldwide scale. However, the introduction of various COVID-19 vaccines, such as those from Pfizer…
Many doctors have thought in the past that age should be an automatic ineligibility factor for multiple myeloma patient transplants. However, Dr. Langren, Dr. Gasparetto, Dr. Landgren, Dr. Hofmeister, and…
According to Pharmaceutical Business Review, Abecma (idecabtagene vicleucel), a CAR T-cell therapy designed to treat patients with relapsed and refractory (R/R) multiple myeloma (MM), received conditional marketing authorization from the…
Drug development is important in creating new therapeutic options for patients with rare or chronic illnesses. One incentivizing program is the Orphan Drug program. Orphan Drug designation is granted to…
KRYPOLIS (carfilzomib) recently received conditional approval in China for the treatment of relapsed/refractory (R/R) multiple myeloma. Licensed by BeiGene, this drug is indicated for adults with R/R multiple myeloma who…
During the European Hematology Association (EHA) Annual Congress (EHA2021), which took place virtually this year, hematological experts and stakeholders discussed new advances in the field. According to Fierce Biotech, one…
Until recently, multiple myeloma patients were reliant upon treatment with immunomodulatory drugs, anti-CD38 antibodies, proteasome inhibitors, and autologous (patient-derived) stem cell transplants. According to a recent article in CancerConnect, patients…
Currently, there are a number of potential treatment options for patients with multiple myeloma: immunomodulators, chemotherapy, HDAC inhibitors, steroids, and proteasome inhibitors. But according to Medical XPress, Peter Mac scientists…
A recent article in Cancer Network announced that motixafortide met its primary endpoint in the GENESIS trial for multiple myeloma patients who were scheduled for stem cell transplantation. Often, the…
In a recent study published in the scientific journal Cell Death & Disease, a novel proteasome inhibitor designated D395 was evaluated as a possible treatment for multiple myeloma, a rare form of…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
March is recognized as Myeloma Action Month, a time dedicated to spreading awareness among the general public and the medical community about multiple myeloma, a rare hematological cancer. This event…
Some cancers and hematological conditions can be difficult to treat. But biotechnology company Oncopeptides AB is working to address this issue for patients with relapsed or refractory (R/R) multiple…
Ionis and the University of California, San Diego (UCSD) have formed a team to investigate antisense oligonucleotide technology as a treatment for multiple myeloma. Their drug, ION251, has shown positive…
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