New Drug/Vaccine Approval

Revuforj™ (revumenib) Tablets – New Drug Approval –November 15, 2024 – The Food and Drug Administration approved revumenib (Revuforj™, Syndax Pharmaceuticals, Inc.), a menin inhibitor, for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older. Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700, NCT04065399; AUGMENT-101) in 104 adult and pediatric patients (at least 30 days old) with relapsed or refractory (R/R) acute leukemia with a KMT2A translocation. <Read More> Kebilidi™ (eladocagene exuparvovec-tneq) Suspension for Intraputaminal Infusion - New Drug Approval – November 13, 2024 – The U.S. Food and Drug Administration approved Kebilidi™ (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi™ is the first FDA-approved gene therapy for treatment of AADC deficiency. <Read More> Aucatzyl™ (obecabtagene autoleucel) Suspension for Intravenous Infusion – New Orphan Drug Approval –November 8, 2024 – The Food and Drug Administration approved obecabtagene autoleucel (Aucatzyl™, Autolus Inc.), a CD19-directed genetically modified autologous T cell immunotherapy, for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Efficacy was evaluated in FELIX (NCT04404660), an open-label, multicenter, single-arm trial that enrolled adults with relapsed or refractory CD19-positive B-cell ALL. Enrolled patients were required to have relapsed following a remission lasting 12 months or less, relapsed or refractory ALL following two or more prior lines of systemic therapy, or disease that was relapsed or refractory 3 or more months after allogeneic stem cell transplantation. <Read More> Danziten™ (nilotinib tartrate) Tablets – New Drug Approval – November 7, 2024 – Azurity Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Danziten™, the first and only nilotinib with no mealtime restrictions indicated for adult patients with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase and adult patients with chronic phase (CP) and acute phase (AP) resistant or intolerant to prior therapy that included imatinib. “Danziten™ offers a new nilotinib treatment option with the equivalent efficacy to Tasigna®, but without the fasting requirements of Tasigna®,” said Richard Blackburn, CEO of Azurity Pharmaceuticals, Inc. <Read More> Emrosi™ (minocycline hydrochloride) Extended-Release Capsules - formerly DFD-29 – New Drug Approval – November 4, 2024 – Journey Medical Corporation (Nasdaq: DERM) (“Journey Medical”), a commercial-stage pharmaceutical company that primarily focuses on selling and marketing U.S. Food and Drug Administration (“FDA”)-approved prescription pharmaceutical products for the treatment of dermatological conditions, announced that the FDA has approved Emrosi™ (Minocycline Hydrochloride Extended Release Capsules, 40 mg), formerly referred to as DFD-29, for the treatment of inflammatory lesions of rosacea in adults. Emrosi™ was developed in collaboration with Dr. Reddy’s Laboratories Ltd. <Read More>

New Indication/Dosage/Formulation Approval

Scemblix® (asciminib) Tablets – New Label Expansion –October 29, 2024 - Novartis announced that Scemblix® (asciminib) was granted accelerated approval by the US Food and Drug Administration (FDA) for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). The accelerated approval is based on major molecular response rate (MMR) at week 48 from the ASC4FIRST Phase III trial that compared once daily Scemblix® to all other investigator-selected (IS) standard of care (SoC) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib, and bosutinib). In the study, Scemblix® demonstrated superior MMR rates in both primary endpoints at week 48 vs. IS SoC TKIs and imatinib alone. Continued approval for the newly diagnosed indication may be contingent upon verification and description of clinical benefit from confirmatory evidence. <Read More> RoxyBond™ (oxycodone hydrochloride) Tablets – New Label Expansion – October 29, 2024 – Protega Pharmaceuticals Inc., an innovative specialty pharmaceutical company focused on responsible pain management and the development of novel abuse-deterrent products, announced that the U.S. Food and Drug Administration (FDA) has approved Roxybond™ (oxycodone hydrochloride) immediate-release (IR) CII 10 mg tablet for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. Roxybond™ is the first and only FDA-approved abuse-deterrent IR 10 mg oxycodone formulation that is expected to reduce abuse by intranasal and intravenous routes. <Read More> Jylamvo™ (methotrexate) Oral Solution – New Label Expansion – October 29, 2024 – Shorla Oncology (‘Shorla’), a U.S.-Ireland specialty pharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has expanded the approval of Jylamvo™ (methotrexate) to include the treatment of pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA). With this approval, Jylamvo™ is currently the only oral liquid methotrexate on the market approved for both adult and pediatric indications. <Read More>

New Drug Shortage

November 18, 2024

• Metronidazole Injection (Currently In Shortage)

November 13, 2024

• Flurbiprofen Tablet (Discontinuation)

• Isosorbide Mononitrate Tablet (Discontinuation)

November 7, 2024

• Aminocaproic Acid Tablet (Discontinuation)

November 4, 2024

• Albuterol Sulfate Syrup (Discontinuation)

• Hydroxocobalamin Injection (Currently In Shortage)

• Ondansetron Hydrochloride Tablet (Discontinuation)

November 1, 2024

• Sodium Bicarbonate Injection (Currently In Shortage)

Updated Drug Shortage

November 18, 2024

• Technetium Tc-99m Sodium Pertechnetate Generator (Resolved)

November 14, 2024

• Penicillin G Benzathine Injection (Currently In Shortage)

October 18, 2024

• Amino Acid Injection (Currently in Shortage)

• Dextrose Monohydrate Injection (Currently in Shortage)

• Lidocaine Hydrochloride Injection (Currently in Shortage)

• Sterile Water Injection (Currently in Shortage)

November 12, 2024

• Bupivacaine Hydrochloride Injection (Currently In Shortage)

• Bupivacaine Hydrochloride, Epinephrine Bitartrate Injection (Currently In Shortage)

• Dextrose Monohydrate 5% Injection (Currently In Shortage)

• Epinephrine Bitartrate, Lidocaine Hydrochloride Injection (Currently In Shortage)

• Hydrocortisone Sodium Succinate Injection (Currently In Shortage)

• Lidocaine Hydrochloride Injection (Currently In Shortage)

• Methotrexate Sodium Injection (Currently In Shortage)

• Methylprednisolone Acetate Injection (Currently In Shortage)

• Penicillin G Benzathine Injection (Currently In Shortage)

• Sodium Chloride 0.9% Injection (Currently In Shortage)

• Sodium Chloride 23.4% Injection (Currently In Shortage)

• Somatropin Injection (Currently In Shortage)

• Sterile Water Injection (Currently In Shortage)

• Technetium Tc-99m Pyrophosphate Kit Injection (Currently In Shortage)

November 7, 2024

• Lactated Ringers Injection (Currently In Shortage)

November 6, 2024

• Bupivacaine Hydrochloride Injection (Currently In Shortage)

• Dexamethasone Sodium Phosphate Injection (Currently In Shortage)

• Dexmedetomidine Hydrochloride Injection (Currently In Shortage)

• Ketamine Hydrochloride Injection (Currently In Shortage)

• Lidocaine Hydrochloride Injection (Currently In Shortage)

• Lorazepam Injection (Currently In Shortage)

• Ropivacaine Hydrochloride Injection (Currently In Shortage)

New Drug Recall and Safety Alerts

Clonazepam Orally Disintegrating Tablets, USP (C-IV) by Endo - Voluntary Recall Expansion – November 18, 2024 – Endo, Inc. (OTCQX: NDOI) (“Endo”) announced that one of its operating subsidiaries, Endo USA, Inc., is expanding its previously announced voluntary recall of Clonazepam Orally Disintegrating Tablets, USP (C-IV) due to potential product carton strength mislabeling. Specifically, Endo’s ongoing investigation has identified the possibility that the Clonazepam product lots listed below contain a limited number of cartons printed with the incorrect strength and National Drug Code (NDC) code due to an error by a third-party packager. The blister strips and tablets inside the product pack reflect the correct strength for the lot. <Read More>

New Generic/Biosimilar Approval and Launch

No new update.

Clinical and Pharmacy News

Biosimilars: Expanding Options for Inflammatory Disease Treatment – November 15, 2024 – Biologics are mainstays of treatment for many chronic inflammatory conditions. Unfortunately, these drugs often come with high price tags due to the significant costs associated with their development and manufacturing. In recent years, several biosimilar products have been developed, with the goal of increasing competition in the biologics market and reducing the cost of care. The FDA defines a biosimilar product as a biologic medication that is highly similar to and has no clinically meaningful differences from an existing FDA-approved biologic. Biosimilars are approved under an abbreviated licensure pathway created by the Biologics Price Competition and Innovation Act of 2009. <Read More> Future of Medicare Drug Price Negotiations Murky Under Trump – November 15, 2024 – The future of one of President Joe Biden’s key domestic policy achievements — getting Medicare to negotiate drug prices — could either become part of the Biden administration’s legacy, get rolled back by the incoming Congress or be weakened by President-elect Donald Trump’s administration. Trump has remained relatively mum about the drug pricing provisions of the 2022 reconciliation bill and its future appears even murkier now that Republicans will control both the House and Senate. “The Trump campaign articulated no position on drug price negotiation specifically, but congressional Republicans are not fans of this program and introduced legislation to repeal the IRA in its entirety,” said Juliette Cubanski, deputy director of the program on Medicare policy at KFF, a health research organization, referring to the law by its nickname, an abbreviation for the Inflation Reduction Act. <Read More> Amazon Introduces Low Upfront Pricing for a Telehealth Visit With Treatment Plan and Fast, Free Medication for Men's Hair Loss, Eyelash Growth, Anti-Aging, ED, and Motion Sickness for Prime Members – November 14, 2024 – Amazon One Medical Pay-per-visit telehealth service offers customers convenient, high-quality health care for 30+ common conditions (such as pink eye, the flu, or a sinus infection) with on-demand messaging visits (where available) for $29 and video visits for $49 payable by credit card or using a Flexible Spending Account or Health Savings Account card. Following the visit, the clinician sends the customer a personalized treatment plan and prescription (if needed), which customers can fulfill at the pharmacy of their choice. Building on this foundation, Amazon is introducing low, clear upfront pricing for a clinical visit, treatment plan, and fast, free medication delivery for Prime members for a range of common health, beauty, and lifestyle concerns, including anti-aging skincare treatment, men’s hair loss, erectile dysfunction, eyelash growth, and motion sickness. <Read More> AI Poised to Improve Trial Recruitment and Drug Adherence in Specialty Pharmacy – November 14, 2024 – The growing potential roles of artificial intelligence, including large-language models and generative AI, were a major topic of discussion at the 2024 NASP Annual Meeting & Expo, in Nashville, Tenn., with applications in clinical trial recruitment and patient engagement among the many uses cited. In specialty pharmacy, AI can potentially enhance patient engagement by personalizing communication and support, leading to improved adherence and satisfaction, suggested Michael Gannon, the senior director of product management for Loopback Analytics. The technology could facilitate clinical trial development by streamlining patient screening, identifying suitable candidates more efficiently and accelerating the approval of new therapies, Mr. Gannon noted. <Read More> New UTI Guidelines Offers Treatment Clarity, Reveals Gaps in Knowledge – November 12, 2024 - New recommendations from the WikiGuidelines Group offer strategies for the prevention, diagnosis, and management of urinary tract infections (UTIs) in children and adults. While the guideline covers a range of clinical topics, including prophylaxis and antimicrobial stewardship, many key clinical questions remain unanswered due to a lack of high-quality evidence, according to lead author Zachary Nelson, PharmD, MPH, of HealthPartners and Park Nicollet Health Services, St. Louis Park, Minnesota, and colleagues. “This guideline fills a critical gap by providing pragmatic, broadly applicable recommendations tailored for generalist care and systems-based practice,” Nelson and colleagues wrote in JAMA Network Open. <Read More> Off-Label Semaglutide Linked to Deaths, Hospitalizations Calling for Compounding Regulation – November 8, 2024 – Approximately 10 deaths and 100 hospitalizations in the United States have been linked to use of off-brand, compounded semaglutide (Ozempic®; Novo Nordisk), according to the drug’s manufacturer Novo Nordisk. The report comes after the company called on the FDA to prohibit compounding of semaglutide in October 2024, citing significant safety concerns around use of off-brand products. “Semaglutide products belong on these lists due to the complexities associated with their formulations, delivery mechanisms, dosage forms, achievement of bioavailability, compounding processes, and physicochemical and analytical testing,” the company stated. “We request that FDA convene and consult an advisory committee on compounding to discuss the addition of semaglutide products to both DDC [Demonstrable Difficulties for Compounding] Lists and promulgate regulations adding semaglutide products to the DDC Lists.” <Read More> How Pharmacy Benefit Managers Are Harming Patients—and What Policymarkers Can Do About It – November 7, 2024 – Earlier this year, the Federal Trade Commission (FTC) announced that it had begun taking legal action against the three largest pharmacy benefit managers (PBMs), the third-party companies that manage prescription drug benefits for health insurance companies and employers. In its filing, the FTC accused these PBMs of abusing their market power to inflate the price of insulin and make it harder for patients to afford it. The three PBMs named by the FTC—Caremark Rx, Express Scripts (ESI), and OptumRx—control about 80 percent of the market. The FTC’s action responds to a very real threat: around 30 percent of the U.S. population struggles to afford prescription drugs, and the burden of unaffordable prescription drug prices does not fall equally. <Read More> Study Finds GLP-1s Safe and Effective for Managing Obesity in Patients with IBD – November 6, 2024 – Glucagon-like peptide-1 receptor agonists are well tolerated and effective for weight loss in patients with inflammatory bowel disease, with a safety profile similar to that observed in the general population prescribed these medications, according to data presented at DDW 2024 by a team of researchers from the University of Pennsylvania. Researchers also saw a reduction in C-reactive protein (CRP) levels, although they said more data are needed to fully explain this change. Experts said this study is particularly relevant given that obesity now affects 15% to 40% of people with IBD (Nat Rev Gastroenterol Hepatol 2017;14[2]:110-121). <Read More>

340B in the News

California Votes to Impose 340B Spending Restrictions on Targeted 340B Providers – November 14, 2024 – California is poised to pass a ballot measure aimed at imposing 340B spending restrictions for certain healthcare entities participating in the 340B Program who have historically engaged in spending that is not directly related to patient care and have also owned or operated multifamily dwellings with significant safety issues. <Read More> J&J Sues Federal Government for Halting 340B Rebate Plan – November 13, 2024 – Johnson & Johnson is suing the Biden administration for blocking a controversial change to how the pharmaceutical company divvies out drug discounts to hospitals in a federal savings program. This summer, J&J unveiled a plan to give hospitals after-the-fact rebates for two medications in the program, called 340B, instead of upfront discounts, but paused implementation following threats of government sanctions. The lawsuit filed Tuesday in a district court seeks a ruling finding J&J’s plan legal and allowing the drugmaker to roll out the model. <Read More>