Monthly Archives: September 2022

Fennec Pharmaceuticals Inc., a specialty pharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has approved Pedmark (sodium thiosulfate injection) to reduce the risk of ototoxicity associated with cisplatin in pediatric patients one month of age and older with localized, non-metastatic solid tumors.

• Pedmark is the first and only FDA-approved therapy Indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients with localized, non-metastatic solid tumors, an area of previously significant unmet medical need.
• The safety and efficacy of Pedmark have not been established when administered following cisplatin infusions longer than 6 Pedmark may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.
• For more information about product availability and patient support, please contact the Fennec HEARS™ program at 1-833-7Pedmark (1-833-773-3627) or visit fennecpharma.com.

Skysona® (elivaldogene autotemcel) is the first FDA approved therapy to slow the progression of neurologic dysfunction with early, active Cerebral Adrenoleukodystrophy (CALD) in boys 4- 17 years of age. Skysona, manufactured by bluebird bio, Inc., is a one-time gene therapy used to treat the underlying cause of CALD.

• It is anticipated that Skysona will be available by the end of 2022 and is a single-dose treatment.
• bluebird bio, has set the wholesale acquisition cost of Skysona in the U.S. at $3.0M per single- dose treatment.

Aponvie® (aprepitant) manufactured by Heron Therapeutics, Inc., a commercial- stage biotechnology company, has been approved for the prevention of postoperative nausea and vomiting (PONV) in adults.

• PONV is commonly experienced after surgery and may result in increased hospital stays, prolonged recovery time, and decreased patient satisfaction.
• Administered via a single 30-second IV injection, Aponvie is the first and only IV formulation of aprepitant for PONV prevention.
• Aponvie should not be used:
  • If you are allergic to aprepitant or any of the ingredients in Aponvie
  • If you are taking pimozide
• The launch date and pricing for Aponvie are pending.

Terlivaz® (terlipressin) has been approved for IV injection. Terlivaz is the first and only FDA-approved product indicated to improve kidney function in adults with hepatorenal syndrome (HRS) with rapid reduction in kidney function.

• HRS involving rapid reduction in kidney function is an acute, life-threatening condition that occurs in people with advanced liver disease.
• Terlivaz is contraindicated:
  • In patients experiencing hypoxia or worsening respiratory symptoms.
  • In patients with ongoing coronary, peripheral, or mesenteric ischemia.
• Terlivaz is expected to be available in the S. in the coming weeks.

Rolvedon™ (eflapegrastim-xnst) injection has been approved to decrease the incidence of chemotherapy-induced febrile neutropenia in adult patients.

• Manufactured by Spectrum Pharmaceuticals, Inc., Rolvedon™ injection is a long-acting granulocyte colony-stimulating factor (G-CSF) with a novel Spectrum has received an indication to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia.
• Rolvedon is contraindicated in patients with a history of serious allergic reactions to eflapegrastim, pegfilgrastim or filgrastim Reactions may include anaphylaxis.
• It is expected to be available in fourth quarter 2022.

Daxxify™ (daxibotulinumtoxin-A-lanm) has been approved as an injection to temporarily improve the look of moderate to severe frown lines (glabellar lines) in adults.

• Revance Therapeutics, Inc. (Nasdaq: RVNC), a biotechnology company focused on innovative aesthetic and therapeutic offerings, announced approval of Daxxify by the FDA.
• Daxxify is the first and only neuromodulator stabilized with Peptide Exchange Technology™ (PXT) and is free of both human serum albumin and animal-based It is due to be available sometime in 2023.
• Daxxify contraindications include hypersensitivity to any botulinum toxin preparation or any of the components in the formulation and infection at the injection site(s).
• Daxxify is not recommended for use in children or pregnant women.

Sotyktu™ (deucravacitinib) has been approved for use as an oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor, for the treatment of moderate to severe plaque psoriasis in adults.

• Manufactured by Bristol-Myers Squibb, Sotyktu is the only approved TYK2 inhibitor worldwide and the first innovation in oral treatment for moderate-to-severe plaque psoriasis in nearly 10 years.
• Sotyktu is not recommended for use in combination with other potent It is currently available at wholesale acquisition cost (WAC) of $6,164.78 for a 30-day supply.

Stimufend® (pegfilgrastim), a biosimilar of Amgen’s Neulasta®, has been approved for use in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with significant incidence of febrile neutropenia.

• Fresenius Kabi, a global health care company, announced FDA approval of its pegfilgrastim biosimilar, Stimufend, for use in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a clinically significant incidence of febrile neutropenia.
• The company expects to launch the product in a prefilled syringe early next year and in an on- body injector.

The U.S. FDA approved Orkambi^® (lumacaftor/ivacaftor) for children with cystic fibrosis ages 1-2 years who have two F508del mutations.

• Manufactured by Vertex Pharmaceuticals Incorporated, Orkambi was previously approved by the FDA for use in people with CF ages 2 years and older with two copies of the 508del mutation.

• This is the third expansion of Orkambi to younger age groups since Orkambi was first approved in 2015 for people with CF ages 12 years and older with two copies of the F508del The drug is one of four medicines approved to treat the faulty CTFR protein, the underlying cause of CF.

The Food and Drug Administration has approved durvalumab (Imfinzi, AstraZeneca UK Limited) in combination with gemcitabine and cisplatin for adult patients with locally advanced or metastatic biliary tract cancer (BTC).

• Imfinzi was originally approved by the FDA, May 1, 2017, for the treatment of adult patients with unresectable, Stage III non-small cell lung cancer (NSCLC) whose disease has not progressed following concurrent platinum-based chemotherapy and radiation therapy.