PDR Drug Update - January 2024

Innovative Therapy Brings Relief to Patients with Rare Soft-Tissue Tumors

Desmoid tumors, sometimes referred to as aggressive fibromatosis, are related to sarcomas and can affect fibrous connective tissues all over the body. The tumors do not have the ability to metastasize, but they can be debilitating and even fatal for patients who experience the condition. Many challenges exist from diagnosis through treatment, and until recently, there have been no FDA-approved therapies for desmoid tumors.

The occurrence of desmoid tumors is generally found in younger individuals aged between 10 and 40 years. No specific cause or trigger has been identified, though there are some possible risk factors, such as being female or having had surgery at the site where a tumor later develops. It is a rare condition, with about 1600 diagnoses occurring annually in the US. In its most recent classifications, the World Health Organization revised its accepted definitions of soft-tissue tumors to support greater precision for applied therapies. Instead of the previous binary categorizations "benign" and "malignant," there now is an "intermediate" category that encompasses the locally aggressive tumor type that is associated with desmoid tumors. Some tumor occurrences do not cause complications for patients and even spontaneously reduce completely. However, these tumors can also grow in such a way that they compress nerves, create intestinal blockages, cause disfigurement, or result in significant pain, impairment, or death.

A more superficial desmoid typically will be less aggressive, while deeper tumors tend to be more active. Depending on the location of a desmoid tumor, its categorization will fall into one of several general groups. Abdominal wall desmoid tumors are found in the upper abdomen's tissues surrounding organs like the stomach. Intra-abdominal or mesenteric tumors are those near the intestinal tract. Lastly are extra-abdominal tumors, encompassing any tumor that develops beyond the abdominal area. The physical locations are usually in the extremities, though the head and neck are also potential spots. The lungs and brain are not generally affected organs, but depending on the nerves and blood vessels involved, any body part may be involved or impacted.

Ultrasound, CT scanning, or MRI is usually used as the first step to diagnose a desmoid tumor. For conclusive diagnosis, a biopsy of the tissue will be conducted to verify the high myofibroblast concentration that is characteristic of the tumors. Earlier management approaches relied more heavily on surgery and chemotherapy, but due to the difficulty of removing the tumors and their tendency to recur with increased complexity, and since neither treatment tack ensures permanent remission, the more recent advisement has been to employ a "wait and see" approach. Now, a new FDA approval for the first drug specifically targeting desmoid tumors is redefining the standard of care for patients with the condition.

Ogsiveo (nirogacestat) has been approved with an indication for adult patients with progressing desmoid tumors who require systemic treatment. It belongs to a new class of drugs, gamma-secretase inhibitors, which can block enzyme activity and thereby restrict Notch protein signaling that is thought to contribute to the growth of desmoid tumors. The results from the phase 3 randomized, controlled study for Ogsiveo showed that individuals treated with the drug were 71% less likely to have died or have their disease worsen than placebo-treated patients. Tumors did not get worse in 76% of nirogacestat-treated patients, compared with 44% of people who received a placebo over a two-year span. Tumor shrinkage was noted in 41% of treated individuals overall. Having a specific drug available like Ogsiveo opens the opportunity for a better quality of life for many patients and sets the stage for further research into optimizing treatment strategies for rare diseases. Stay informed about critical drug information, including drug approvals for ultra-rare diseases, by exploring PDR by ConnectiveRx. You can also stay current by using the official PDR app, mobilePDR, which is available for free from your preferred app store.

FDA New Approvals

Avzivi

The FDA has approved Avzivi (bevacizumab-tnjn), a biosimilar referencing Avastin. Avzivi is a humanized monoclonal antibody that targets VEGF. Learn more about Avzivi

Casgevy

The FDA has approved Casgevy (exagamglogene autotemcel), a cell-based gene therapy, for the treatment of sickle cell disease in patients aged 12 years and older with recurrent vaso-occlusive crises. Learn more about Casgevy

Fabhalta

The FDA has approved Fabhalta (iptacopan) as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria. Learn more about Fabhalta

Filsuvez

The FDA has approved Filsuvez (birch triterpenes) topical gel for the treatment of partial thickness wounds in patients aged 6 months and older with junctional epidermolysis bullosa and dystrophic epidermolysis bullosa. Learn more about Filsuvez

iDose TR

The FDA has approved iDose TR (travoprost) intracameral implant, a prostaglandin analog indicated for the reduction of intraocular pressure in patients with ocular hypertension or open-angle glaucoma. Learn more about iDose TR

Iwilfin

The FDA has approved Iwilfin (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-GD2 immunotherapy. Learn more about Iwilfin

Lyfgenia

The FDA has approved Lyfgenia (lovotibeglogene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years or older with sickle cell disease and a history of vaso-occlusive events. Learn more about Lyfgenia

Tarpeyo

The FDA has approved Tarpeyo (budesonide) delayed-release capsules to reduce the loss of kidney function in adults with primary immunoglobulin A nephropathy at risk for disease progression. Learn more about Tarpeyo

Wainua

The FDA has approved Wainua (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Learn more about Wainua

FDA Labeling Changes

Brukinsa

The FDA has approved a label update for Brukinsa (zanubrutinib) to include superior progression-free survival results from the Phase 3 ALPINE trial comparing Brukinsa against Imbruvica (ibrutinib) in previously treated patients with relapsed or refractory chronic lymphocytic leukemia. Learn more about Brukinsa

Carvykti

The FDA issued a change to the warning label for Carvykti (ciltacabtagene autoleucel). The boxed warning on the label includes a new item stating that secondary hematological malignancies, including myelodysplastic syndrome and acute myeloid leukemia, have occurred following treatment with Carvykti. Learn more about Carvykti

FDA New Indications

Cresemba

The FDA has approved Cresemba (isavuconazonium sulfate), an azole antifungal drug, for the treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in pediatric patients. Cresemba for injection is approved for adults and now for pediatric patients aged 1 year and older. Cresemba capsules are approved for adults and now for pediatric patients aged 6 years and older who weigh 16kg and greater. Learn more about Cresemba

Welireg

The FDA has approved Welireg (belzutifan) for the treatment of adult patients with advanced renal cell carcinoma following a programmed death receptor-1 or programmed death-ligand 1 inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor. Learn more about Welireg

Zoryve

The FDA has approved Zoryve (roflumilast) topical foam, 0.3%, for the treatment of seborrheic dermatitis in individuals aged 9 years and older. Learn more about Zoryve

FDA Recalls

Hospira Bleomycin

Hospira, Inc. is voluntarily recalling one lot of bleomycin for injection, USP 15 units single dose Onco-Tain glass flip-top vial, lot BL12206A, to the user level. The recall was initiated due to a confirmed customer report for the presence of glass particulate within a single vial. Learn more about the Hospira bleomycin recall

Insight Pharmaceuticals Benzocaine

Insight Pharmaceuticals is voluntarily recalling lot 1A16420 of Americaine 20% benzocaine topical anesthetic spray, to the consumer level. A recent review found that a sample showed a low level of benzene came from the propellant that sprays the product out of the can. Learn more about the Insight Pharmaceuticals benzocaine recall

InvaGen Pharmaceuticals Vigabatrin

InvaGen Pharmaceuticals is voluntarily recalling vigabatrin for oral solution, USP 500mg, lot NB301030, to the consumer level. The product has been found to have seal integrity issues, allowing for powder leakage from the pouch. Learn more about the InvaGen Pharmaceuticals vigabatrin recall

January 2024