Abbvie Inc., of North Chicago

Skyrizi (risankizumab)

IL-23 inhibitor

Moderate to severe plaque psoriasis

EMA's Committee for Medicinal Products for Human Use recommended approval for use in adults who are candidates for systemic therapy

Adlon Therapeutics LP, of Stamford, Conn., a subsidiary of Purdue Pharma LP

Adhansia XR (methylphenidate hydrochloride) extended-release capsules CII

CNS stimulant

Attention deficit hyperactivity disorder

Approved by FDA for use in patients 6 and older

Akcea Therapeutics Inc., of Boston, an affiliate of Ionis Pharmaceuticals Inc.

Waylivra (volanesorsen)

Reduces production of ApoC-III

Familial chylomicronemia syndrome

EMA's Committee for Medicinal Products for Human Use recommended conditional approval as an adjunct to diet in adults with genetically confirmed FCS who are at high risk for pancreatitis, in whom response to diet and triglyceride-lowering therapy has been inadequate

Ascendis Pharma A/S, of Copenhagen

Transcon CNP

Long-acting prodrug of C-type natriuretic peptide

Achondroplasia

FDA granted orphan drug designation

Astellas Pharma Inc., of Tokyo

Xospata (gilteritinib)

Small-molecule FLT3 inhibitor

Relapsed/refractory acute myeloid leukemia

MAA accepted for review by EMA; seeks approval for use in adults whose disease carries FLT3 mutation

Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J.

Lynparza (olaparib)

PARP inhibitor

Breast cancer

EMA's Committee for Medicinal Products for Human Use adopted a positive opinion, recommending approval as a monotherapy to treat adults with germline BRCA1/2 mutations who have HER2-negative locally advanced or metastatic disease

Biomarin Pharmaceutical Inc., of San Rafael, Calif.

Palynziq (pegvaliase)

Substitutes deficient phenylalanine hydroxylase enzyme

Phenylketonuria

EMA's Committee for Medicinal Products for Human Use recommended approval for use in patients, 16 and older, who have inadequate blood Phe control (blood Phe levels greater than 600 micromol/L) despite prior management with available treatment options

Carsgen Therapeutics Ltd., of Shanghai

CT-053

Fully human BCMA CAR T-cell candidate

Relapsed/refractory multiple myeloma

Received IND clearance from China's National Medical Products Administration

Glaxosmithkline plc, of London

Dectova (zanamivir)

Antiviral

Complicated and potentially life-threatening influenza A or B virus

EMA's Committee for Medicinal Products for Human Use recommended approval for use in adults and pediatric patients, ages ≥6 months when influenza virus is known or suspected to be resistant to agents other than zanamivir, and/or other antiviral agents, including inhaled zanamivir (Relenza), are not suitable for the individual patient

Hansa Biopharma AB, of Lund, Sweden

Idefirix (imlifidase)

Enzyme cleaving IgG

Treatment to enable kidney transplantation in highly sensitized patients

EMA accepted the MAA for review

Novo Nordisk A/S, of Bagsvaerd, Denmark

Fiasp (insulin aspart injection) 100 u/ML

Insulin

Type 1 diabetes

Filed for label updates to the EMA and the FDA seeking approval for use as a mealtime insulin for children and adolescents

Outlook Therapeutics Inc., of Cranbury, N.J.

ONS-5010

Ophthalmic formulation of anti-VEGF antibody bevacizumab

Wet age-related macular degeneration

Submitted IND to FDA to begin enrolling patients in the U.S. portion of the ONS-5010-002 phase III trial

Pfizer Inc., of New York

Lorviqua (lorlatinib)

Tyrosine kinase inhibitor inhibiting ALK

ALK-positive advanced non-small-cell lung cancer

EMA's Committee for Medicinal Products for Human Use recommended conditional marketing authorization as a monotherapy for patients whose disease has progressed after alectinib or ceritinib as the first ALK TKI therapy, or crizotinib and at least 1 other ALK TKI; conversion to normal approval will be contingent on provisions of comprehensive data confirming benefit-risk

Portola Pharmaceuticals Inc., of South San Francisco

Ondexxya (andexanet alfa)

Coagulation factor Xa (recombinant), inactivated

Reversal of anticoagulant effects of factor Xa inhibitors apixaban or rivaroxaban

EMA's Committee for Medicinal Products for Human Use adopted a positive opinion on the MAA seeking approval for use in patients experiencing uncontrolled or life-threatening bleeding

Sanofi SA, of Paris, and Lexicon Pharmaceuticals Inc., of The Woodlands, Texas

Zynquista (sotagliflozin)

Oral dual SGLT1/2 inhibitor

Type 1 diabetes mellitus

EMA's Committee for Medicinal Products for Human Use recommended approval in both a 200-mg and 400-mg dose for use as adjunct to insulin to improve glycemic control in adults with body mass index of ≥ 27 kg/m2, who have failed to achieve adequate glycemic control despite optimal insulin therapy

Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y.

Dupixent (dupilumab)

IL-4/IL-13-inhibiting monoclonal antibody

Severe asthma

EMA's Committee for Medicinal Products for Human Use recommended approval as an add-on maintenance treatment for severe asthma with type 2 inflammation in adults and adolescents 12 and older

Notes

For more information about individual companies and/or products, see Cortellis.