FDA Approves Imkeldi Oral Solution for the Treatment of Certain Forms of Leukemia and Other Cancers

• Shorla Oncology has announced that the U.S. Food and Drug Administration (FDA) has approved Imkeldi (imatinib) oral solution, to treat certain forms of leukemia and other cancers, and it is designed to provide dosing accuracy.
• Imkeldi can help slow or prevent the growth of specific cancers, including chronic myeloid leukemia (CML) and acute lymphoblastic leukemia, myelodysplastic syndrome /myeloproliferative disease (MDS/MPD), and gastrointestinal tumors (GIST).
• Imatinib mesylate is a tyrosine kinase inhibitor, approved by the FDA for use in certain forms of leukemia (such as acute lymphoblastic leukemia and chronic myeloid leukemia) and other cancers in adults and pediatric patients as young as one year old. Imatinib is available both generically and as a brand name, Gleevec tablets, which were approved in 2001.

Source: Shorla Oncology

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FDA Approves Attruby to Reduce Cardiovascular Death and Cardiovascular-Related Hospitalization in Patients with ATTR-CM

BridgeBio Pharma, Inc. has announced that the U.S. Food and Drug Administration (FDA) approved Attruby™ (acoramidis), for the treatment of adults with cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization.

The FDA approval is based on positive results seen in the ATTRibute-CM Phase 3 study, where Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life.

• Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. It mimics a naturally occurring “rescue mutation” of the TTR gene that targets the root cause of ATTR-CM.
• Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.

Source: BridgeBio Pharma, Inc.

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FDA Grants Accelerated Approval to Ziihera for the Treatment of HER2-Positive Biliary Tract Cancer

Jazz Pharmaceuticals plc has announced the U.S. Food and Drug Administration (FDA) accelerated approval of Ziihera® (zanidatamab-hrii) 50mg/mL for injection for intravenous use for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. Ziihera was approved under accelerated approval based on a 52% objective response rate (ORR) and a median duration of response (DOR) of 14.9 months as determined by independent central review (ICR).

• Continued approval for this indication may be contingent upon verification and description of
  clinical benefit in a confirmatory trial.
• Biliary Tract Cancer, including gallbladder cancer and intrahepatic and extrahepatic
  cholangiocarcinoma, account for <1% of all adult cancers globally and are often associated with a
  poor prognosis.

Source: Jazz Pharmaceuticals plc

FDA Approves Revuforj for the Treatment of Adult and Pediatric Patients with Relapsed or Refractory Acute Leukemia with a KMT2A Translocation

Syndax Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Revuforj (revumenib) as the first and only menin inhibitor for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older. The FDA previously granted Breakthrough Therapy and Fast Track designations as well as Priority Review for Revuforj. The New Drug Application (NDA) received approval through the FDA’s Real Time Oncology Review (RTOR) program.

• Syndax expected that the 110 and 160mg tablets of Revuforj will be available for order in the United States through a network of specialty distributors and specialty pharmacies in November. Syndax expected that the 25mg tablets, which may be used to treat patients who weigh less than 40kg, will be commercially available in the late first quarter or early second quarter of 2025.
• Prior to commercial availability of the 25mg tablets, an oral solution of revumenib will be available through an expanded access program to allow for dosing of patients who weigh less than 40kg.

Source: Syndax Pharmaceuticals, Inc.

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FDA Approves Danziten Tablets, the First and Only Nilotinib with No Mealtime Restrictions

Azurity Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Danziten. This is 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 in adult patients with chronic phase (CP), and acute phase (AP) resistant or intolerant to prior therapy that included imatinib.

• Tasigna® has established efficacy in adults with newly diagnosed Ph+ CML-CP and resistant or
  intolerant Ph+ CML-CP and CML-AP; however, Tasigna has variable bioavailability that considerably
  increases when taken with food.
• Danziten offers a new nilotinib treatment option with the equivalent efficacy to Tasigna, but
  without the fasting requirements of Tasigna.

Source: Azurity Pharmaceuticals, Inc.

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FDA Approves Kebilidi Gene Therapy for the Treatment of AADC Deficiency

PTC Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain.

• The gene therapy, which will be marketed in the United States with the brand name Kebilidi™ (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity.
• AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. Kebilidi is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure.

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FDA Approves Aucatzyl for Adults with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

Autolus Therapeutics plc has announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Aucatzyl (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).

• Aucatzyl is a CD19-directed genetically modified autologous T cell immunotherapy.
• Aucatzyl is the first CAR T therapy approved by the FDA with no requirement for a REMS program (Risk Evaluation Mitigation Strategy).
• ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system, and other organs.

Source: Autolus Therapeutics plc

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November 2024

Exenatide Injection 300mcg/1.2mL (250mcg/mL) and 600mcg/2.4mL (250mcg/mL)
Approved: November 19, 2024 – Amneal Pharmaceuticals LLC
Treatment for: Type 2 Diabetes
Generic for: Byetta

July 2024

• Indium In-111 Pentetreotide Injection Kit 3 mCi/mL
  Approved: July 1, 2024 – Sun Pharmaceutical Industries, Inc.
  Used for: Diagnosis and Investigation
  Generic for: Octreoscan
• Bupivacaine Liposome Injectable Suspension 133mg/10mL (13.3mg/mL) and 266mg/20mL
  (13.3mg/mL)
  Approved: July 1, 2024 – Jiangsu Hengrui Pharmaceuticals Co., Ltd.
  Used for: Analgesia
  Generic for: Exparel

June 2024

• Palbociclib Tablets 75mg, 100mg and 125mg
  Approved: June 5, 2024 – Synthon Pharmaceuticals, Inc.
  Treatment for: Breast Cancer
  Generic for: Ibrance Tablets
• Avanafil Tablets 50mg, 100mg and 200mg
  Approved: June 14, 2024 – Hetero Labs Limited
  Treatment for: Erectile Dysfunction
  Generic for: Stendra
• Phentermine Hydrochloride and Topiramate Extended-Release Capsules 3.75mg (base)/23mg,
  7.5mg (base)/46mg, 11.25mg (base)/69mg and 15mg (base)/92mg
  Approved: June 25, 2024 – Actavis Laboratories FL, Inc.
  Treatment for: Obesity
  Generic for: Qsymia