2024 Approvals

Basilea: ZEVTERA antibiotic
Basilea Pharmaceutica received approval from the U.S. FDA for ZEVTERA (ceftobiprole medocaril sodium for injection), for the treatment of adult patients with Staphylococcus aureus bloodstream infections, including right-sided infective endocarditis, and adults with acute bacterial skin and skin structure infections (ABSSSI) and for adult and pediatric patients with community-acquired bacterial pneumonia (CABP).
 
The NDA was supported by clinical efficacy and safety data from phase 3 studies ERADICATE (SAB)1 and TARGET (ABSSSI), and a phase 3 study in CABP. The ERADICATE study was the largest double-blind randomized registrational study conducted for a new antibiotic treatment in SAB.

Vanda Pharmaceuticals: Fanapt in Acute Bipolar I Disorder
Vanda Pharmaceuticals Inc. received approval from the FDA for Fanapt (iloperidone) tablets for the acute treatment of manic or mixed episodes associated with bipolar I disorder in adults. Fanapt is an atypical antipsychotic agent that has been used for the acute treatment of patients with schizophrenia since its FDA approval
in 2009.
 
The approval was based on a pivotal study randomizing approximately 400 patients. The study showed Fanapt treated patients showed a larger improvement than placebo treated patients, and this difference was highly statistically significant. YMRS was assessed at the end of Weeks 1, 2, 3 and 4. Statistically significant benefit in the Fanapt treated group over placebo was observed as early as the Week 2 assessment.

AstraZeneca: Ultomiris in NMOSD
AstraZeneca’s Ultomiris (ravulizumab-cwvz) has been approved by the U.S. FDA as a long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval was based on positive results from the CHAMPION-NMOSD Phase III trial.
 
NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves.

AbbVie: ELAHERE in Ovarian Cancer
The U.S. FDA has granted full approval to AbbVie for ELAHERE (mirvetuximab soravtansine-gynx) for the treatment of folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube or primary peritoneal adult cancer patients treated with up to three prior therapies.
 
Patients with these cancers often present with late-stage disease, undergo surgery and are then treated with platinum-based chemotherapy. They may become resistant to this treatment and require another therapy, such as ELAHERE.
 
AbbVie added ELAHERE to its portfolio recently when it completed its acquisition of ImmunoGen.

Italfarmaco: Duvyzat in Duchenne muscular dystrophy
The U.S. FDA has approved Italfarmaco S.p.A.’s Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor, for the treatment of patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare X-linked progressive and life-limiting neuromuscular condition with symptoms from early childhood.
 
The approval is based on the results of the pivotal multicenter, randomized, double-blind, placebo-controlled phase 3 EPIDYS trial (NCT02851797).

Idorsia: TRYVIO in Hypertension
The U.S. FDA has approved Idorsia Ltd.’s TRYVIO (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs. 
 
TRYVIO (aprocitentan) is an endothelin receptor antagonist that inhibits the binding of endothelin (ET)-1 to ETA and ETB receptors. The effects of ET-1 bear many similarities with the pathophysiology of hypertension, and ET-1 is a major driver of aldosterone production.
 
TRYVIO was evaluated as a monotherapy in a Phase 2 study in patients with hypertension, and as an add-on therapy in a Phase 3 study called PRECISION in patients with confirmed resistant hypertension. In PRECISION, aprocitentan was well tolerated and superior to placebo in lowering blood pressure at week 4, with a sustained effect at week 40.

Allecra Therapeutics: EXBLIFEP in Urinary Tract Infections
Allecra Therapeutics, a biopharmaceutical company developing therapies to combat antibiotic resistance, received approval from the FDA for EXBLIFEP (cefepime/enmetazobactam), as a treatment for complicated urinary tract infections (cUTIs), including pyelonephritis, in patients 18 years and older. Allecra has also received a five-year marketing exclusivity extension from the FDA as part of the Generating Antibiotic Incentives Now Act (GAIN Act). The GAIN Act, enacted by the U.S. Congress, incentivizes the creation of new anti-infective therapeutics by providing benefits to manufacturers of Qualified Infectious Disease Products (QIDPs).
 
The approval of EXBLIFEP was supported by clinical data that demonstrated its effectiveness against antimicrobial resistance in gram-negative bacteria, especially resistance mediated by both ESBL (Extended Spectrum Beta Lactamases) and AmpC. This included results from Allecra’s Phase 3 ALLIUM trial, which met criteria for non-inferiority and superiority compared to piperacillin/tazobactam in the primary composite outcome of clinical cure and microbiological eradication in patients with cUTIs.
 
Novartis: Xolair in Pediatric Food Allergies 
Novartis received approval from the FDA for Xolair (omalizumab) for the reduction of allergic reactions, including anaphylaxis, that may occur with accidental exposure to one or more foods in adult and pediatric patients aged 1 year and older with IgE-mediated food allergy. People taking Xolair for food allergies should continue to avoid all foods they are allergic to. Xolair should not be used for the emergency treatment of any allergic reactions, including anaphylaxis. Immunoglobulin E (IgE)-mediated food allergies are the most common type and are typically characterized by the rapid onset of symptoms following exposure to certain food allergens. Xolair is the first and only FDA-approved medicine to reduce allergic reactions in people with one or more food allergies. 
 
Approval is based on data from the NIH-sponsored Phase III OUtMATCH study, which showed a significantly higher proportion of food allergy patients as young as 1 year treated with Xolair could tolerate small amounts of peanut, milk, egg and cashew without an allergic reaction, compared to placebo.
 
Teva, Alvotech: SIMLANDI biosimilar to Humira
Teva and Alvotech received approval from the FDA for SIMLANDI (adalimumab-ryvk) injection as an interchangeable biosimilar to AbbVie’s Humira, for the treatment of adult rheumatoid arthritis, juvenile idiopathic arthritis, adult psoriatic arthritis, adult ankylosing spondylitis, Crohn’s disease, adult ulcerative colitis, adult plaque psoriasis, adult hidradenitis suppurativa and adult uveitis.
 
SIMLANDI is the first high-concentration, citrate-free biosimilar to Humira that has been granted an interchangeability status by the FDA, and will qualify for interchangeable exclusivity for the 40mg/0.4ml injection. In the U.S. today, nearly 88% of U.S. prescriptions for adalimumab are for the high-concentration presentation.
 
Iovance Biotherapeutics: AMTAGVI in Melanoma
Iovance Biotherapeutics, Inc., a biotechnology company developing polyclonal tumor infiltrating lymphocyte (TIL) cell therapies for cancer, received approval from the FDA for AMTAGVI (lifileucel) suspension for intravenous infusion. AMTAGVI is a tumor-derived autologous T cell immunotherapy for the treatment of adults with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. This indication is approved under an accelerated approval based on overall response rate (ORR) and duration of response. Iovance is also conducting TILVANCE-301, a Phase 3 trial to confirm clinical benefit.
 
AMTAGVI is the first and the only one-time, individualized T cell therapy to receive FDA approval for a solid tumor cancer. The proposed mechanism for AMTAGVI offers a new cell therapy approach that deploys patient-specific T cells called TIL cells. When cancer is detected, the immune system creates TIL cells to locate, attack, and destroy cancer. 
 
The approval is based on results from the C-144-01 clinical trial, a global, multicenter trial investigating AMTAGVI in patients with advanced melanoma previously treated with anti-PD-1 therapy and targeted therapy. The primary efficacy analysis included 73 patients who received the recommended AMTAGVI dose. Among the 73 patients, 31.5% achieved an objective response by Response Evaluation Criteria in Solid Tumors with a median duration of response not reached at 18.6 months follow-up (43.5% of responses had a duration greater than 12 months).
 
Sanofi: Dupixent in Eosinophilic Esophagitis 
Sanofi received approval from the FDA for Dupixent (dupilumab) for the treatment of pediatric patients aged 1 to 11 years with eosinophilic esophagitis (EoE). Dupixent is now the first and only medicine approved in the U.S. specifically indicated to treat these patients. 
 
This approval expands the initial FDA approval for EoE in May 2022 for patients aged 12 years and older. The FDA evaluated Dupixent for this expanded indication under Priority Review.
 
EoE is a chronic, progressive disease driven in part by type 2 inflammation that damages the esophagus and impairs its function. EoE can severely impact a child’s ability to eat, and they may experience heartburn, vomiting, abdominal discomfort, trouble swallowing, food refusal and failure to thrive. Approximately 21,000 children under the age of 12 in the U.S. are currently being treated for EoE with unapproved therapies. 
 
Takeda: GAMMAGARD LIQUID in CIDP
Takeda received approval from the FDA for GAMMAGARD LIQUID [Immune Globulin Infusion (Human) 10% solution] as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). It can be used as induction therapy, which includes an induction dose followed by maintenance doses. 
 
The approval is based on results from an open-label, single-arm, multicenter clinical study (ADVANCE-CIDP 2) that evaluated the efficacy and safety of GAMMAGARD LIQUID in adults with CIDP who developed a relapse. Efficacy in the study was based on responder rate, defined as a subject who demonstrated an improvement of functional disability. The responder rate was 94%. Improvement in grip strength and change in Rasch-built Overall Disability Scale (R-ODS) score were recorded across participants.
 
CIDP is a rare, acquired, immune-mediated neuromuscular disorder affecting the peripheral nervous system. It’s characterized by progressive, symmetric symptoms such as weakness, tingling or loss of feeling in distal and proximal limbs, loss of reflexes and difficulty walking. The mechanism of action of immunoglobulins in the treatment of CIDP in adults has not been fully elucidated but may include immunomodulatory effects.

Filings

Bayer: BAY 2927088 in NSCLC
Bayer was granted Breakthrough Therapy designation from the FDA for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy.
 
BAY 2927088 is an oral, reversible tyrosine kinase inhibitor (TKI) that potently inhibits mutant human epidermal growth factor receptors 2 (HER2), including HER2 exon 20 insertions and HER2 point mutations, as well as epidermal growth factor receptors (EGFR), with high selectivity for mutant vs wild-type EGFR.
 
Lung cancer is the leading cause of cancer-related deaths worldwide,3 and NSCLC is the most common type of lung cancer, accounting for more than 85% of cases. Activating HER2 mutations are found in 2% to 4% of advanced NSCLC. Currently there are no available therapies that have received full approval in the U.S. for patients with NSCLC in the metastatic or advanced setting harboring HER2 activating mutations.

NeoImmuneTech: NT-I7 in Pancreatic Cancer
NeoImmuneTech, Inc., a T cell-focused therapeutics company, was granted Orphan Drug Designation (ODD) from the FDA for NT-I7 (efineptakin alfa) (rhIL-7-hyFc) for the treatment of pancreatic cancer.
 
NT-I7 has been studied in several phase I and II clinical trials and has demonstrated the potential to amplify T cells across the subsets, boost the immune system, and enhance the anti-tumor response in people with pancreatic cancer and other solid tumors.
 
Pancreatic cancer is an aggressive tumor-type associated with extremely poor prognosis. It is the third deadliest cancer in the U.S. and EU with a combined annual mortality of 139,000.