PDR Drug Update - February 2025

Non-Factor Treatment for Hemophilia

The nature of hemophilia and the complexity of its treatments translate into a life complicated with varying limitations for patients. In order to avoid risks that could result in dangerous hemorrhaging, extensive lifestyle modification is needed. The burden of disease is significant and adversely affects patients and caregivers in many ways. A new FDA approval for a novel medication for the treatment of hemophilia may offer relief of some of that burden.

Hemophilia types A and B are rare bleeding disorders where patients’ blood is not able to clot properly. The condition occurs because these patients have deficiencies of specific proteins called clotting factors. When there are missing or lower levels of clotting factor VIII (FVIII), it is referred to as hemophilia A. When the lacking protein is factor IX (FIX), it is called hemophilia B. Patients with either form of hemophilia may experience bruising on the skin and bleeding from wounds that continues excessively. There is also the possibility of spontaneous bleeding inside soft tissues, muscles, and joints. This internal bleeding can lead to significant pain and stiffness and can permanently damage joints.

The general approach to managing bleeding episodes involves either episodic treatment administered on-demand or prophylactic treatment using medication containing FVIII or FIX or a product that mimics the appropriate clotting factor. Examples of treatment medications include Hemlibra (emicizumab-kxwh), Stimate (desmopressin acetate), and Hemgenix (etranacogene dezaparvovec-drlb). The reality for many patients with hemophilia is that management of the condition involves regular intravenous infusions, often requiring administration multiple times each week and sometimes requiring trips to a hemophilia treatment center. Typically, infusions are given through the arm or a port in the chest. When the clotting factors are restored, patients’ systems can resume normal clotting functions temporarily.

This current standard of care does help reduce the risk of fatal bleeding events and serious joint damage from bleeding for a great deal of patients. Adding to the available methods is a recent FDA approval using an innovative mechanism that targets the body’s natural tissue factor pathway inhibitor (TFPI). It provides patients with an alternative treatment approach that addresses some of the ongoing challenges stemming from hemophilia A and B. The novel treatment option is Hympavzi (marstacimab-hncq), a human monoclonal antibody that inhibits the anticoagulation activity of TFPI. As it accomplishes this function, the availability of free factor Xa (a protein in blood involved in the coagulation cascade) is increased, allowing for additional thrombin generation. Hemostasis is promoted, and the body is able to form clots, reducing issues with excess bleeding. Bypassing the need to supplement FVIII or FIX may be desirable for treatment in certain patients with hemophilia.

Hympavzi’s prescribing information describes its indication as being for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors or hemophilia B (congenital factor IX deficiency) without factor IX inhibitors. It is administered once weekly via a subcutaneous injection using a prefilled autoinjector pen and can be self-injected or given by a caregiver. This flexibility offers relief for patients who previously may have needed more frequent and time-consuming dosing. Hympavzi’s labeling includes warnings and precautions about the potential for thromboembolic events, hypersensitivity concerns, and embryofetal toxicity. Close monitoring and advisement from the healthcare team managing patients with hemophilia is needed. Continued study for the therapy is underway, with the inhibitor cohort of its clinical trial still ongoing. Results of the study are expected later this year. Stay informed about emergent drug information, including treatment options for hemophilia, 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

Datroway

The FDA has approved Datroway (datopotamab deruxtecan) for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease. Learn more about Datroway

Journavx

The FDA has approved Journavx (suzetrigine), an oral, non-opioid, highly selective NaV1.8 pain signal inhibitor, for the treatment of adults with moderate-to-severe acute pain. Learn more about Journavx

Symbravo

The FDA has approved Symbravo (meloxicam/rizatriptan) for the acute treatment of migraine with or without aura in adults. Learn more about Symbravo

FDA New Indications

Calquence

The FDA has approved Calquence (acalabrutinib) in combination with bendamustine and rituximab for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are ineligible for autologous hematopoietic stem cell transplantation. This approval additionally converts Calquence's accelerated approval to a full approval for adult patients with MCL treated with at least one prior therapy, as granted by the FDA in October 2017. Learn more about Calquence

Enhertu

The FDA has approved Enhertu (trastuzumab deruxtecan) for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, that has progressed on one or more endocrine therapies in the metastatic setting. Learn more about Enhertu

Lumakras

The FDA has approved Lumakras (sotorasib) in combination with Vectibix (panitumumab) for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer, as determined by an FDA-approved test, who have received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Learn more about Lumakras

Omvoh

The FDA has approved Omvoh (mirikizumab-mrkz) for the treatment of moderately to severely active Crohn's disease in adults. Learn more about Omvoh

Ozempic

The FDA has approved Ozempic (semaglutide) for reducing the risk of kidney disease worsening, kidney failure (end-stage kidney disease), and death due to cardiovascular disease in adults with type 2 diabetes and chronic kidney disease. Learn more about Ozempic

Spravato

The FDA has approved Spravato (esketamine) nasal spray as monotherapy for adults living with major depressive disorder who have had an inadequate response to at least two oral antidepressants. Learn more about Spravato

FDA Recalls

Alvogen Fentanyl Transdermal System

Alvogen, Inc. is voluntarily recalling one lot of Fentanyl Transdermal System 25mcg/h transdermal patches to the consumer level. The reason for the recall is that there is a potential that patches could be multi-stacked, adhered one on top of the other, in a single product pouch. Learn more about the Alvogen Fentanyl Transdermal System recall

Provepharm Phenylephrine Hydrochloride

Provepharm Inc. is voluntarily recalling lot number 24020027, expiry date December 2025, of phenylephrine hydrochloride injection, USP, 10mg/mL (pharmacy bulk package) at the hospital/institutional level. This recall was initiated based on a customer complaint from a pharmacy after observing a visible black particulate matter found in a single-sealed vial of the product. Learn more about the Provepharm phenylephrine hydrochloride recall

FDA Alerts

Anaphylaxis with Glatiramer Acetate

The FDA is warning about the risk of a rare but serious allergic reaction with the medicine glatiramer acetate (Copaxone, Glatopa). This serious allergic reaction, called anaphylaxis, can occur at any time while on treatment, after the first dose, or after doses administered months or years after starting the medicine. Learn more about the glatiramer acetate drug safety communication

Epinephrine from BPI Labs and Endo USA

The FDA is warning healthcare professionals not to use unapproved epinephrine nasal solutions manufactured by BPI Labs LLC and Endo USA. Healthcare professionals have confused these products with FDA-approved injectable epinephrine products for intravenous use. BPI Labs and Endo USA nasal solutions products should never be injected intravenously. Learn more about epinephrine from BPI Labs and Endo USA

GBS Warning in Prescribing Information for RSV Vaccines

The FDA has required and approved safety labeling changes to the Prescribing Information for Abrysvo (respiratory syncytial virus vaccine) and Arexvy (respiratory syncytial virus vaccine, adjuvanted) due to observational study suggesting an increased risk of Guillain-Barré syndrome (GBS) during the 42 days following vaccination with a respiratory syncytial virus (RSV) vaccine. Learn more about the GBS drug safety communication

February 2025