Specialty Drug Pipeline Monthly Update: October 2023

Critical updates in an ever-changing environment

October 30, 2023
This monthly pipeline wrap-up provides a review of newly approved specialty drugs, recent drug launches, generic updates, new indications and news of note on drugs in the approval process.

See separate article for pipeline information on traditional drugs.

New Drug Information

  • Tofidence™ (tocilizumab-bavi): The United States (U.S.) Food and Drug Administration (FDA) approved Tofidence, by Biogen and Bio-Thera Solutions as the first biosimilar to Genentech’s Actemra® (tocilizumab). The interleukin-6 (IL-6) receptor antagonist is indicated for the treatment of adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response  to at least 1 disease-modifying antirheumatic drug (DMARD) and patients 2 years and older with polyarticular JIA (pJIA) or systemic JIA (sJIA).[i] Biosimilars are highly similar to and have no clinically meaningful differences from its reference product. Tofidence was approved as a 20mg/mL preservative-free solution for further dilution before intravenous (IV) infusion. Unlike Actemra, Tofidence is not approved for giant cell arteritis (GCA), systemic sclerosis-associated interstitial lung disease (SSc-ILD), cytokine release syndrome (CRS), or Coronavirus disease 2019 (COVID-19). Both Tofidence and Actemra have a boxed warning for risk of serious infections. Launch timeframe of Tofidence has not been announced. Pricing to follow launch.
  • Rivfloza™ (nedosiran): Rivfloza is an lactate dehydrogenase A (LDHA)-directed small interfering RNA (siRNA) agent. It was granted Orphan Drug and Rare Pediatric Disease designations and approved by the FDA to lower urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function (e.g., estimated glomerular filtration rate [eGFR] ≥ 30 mL/min/1.73 m2).[ii] Rivfloza is administered as a once-monthly subcutaneous (SC) injection by the patient or caregiver. In the phase 2 PHYOX2 (NCT03847909) clinical trial, Rivloza led to a marked reduction from baseline in 24 hour-urinary oxalate (Uox) excretion from Day 90 to Day 180 compared to placebo (LS mean percent change from baseline, -37% versus +12%; respectively). Rivfloza is the second siRNA agent FDA-approved to treat PH1; following Oxlumo® (lumasiran; Alnylam), which targets hydroxyacid oxidase 1 (HAO1) and is administered by a healthcare professional via monthly SC injections. Rivfloza launch is anticipated in early 2024, with pricing to follow.
  • Entyvio® SC (vedolizumab): A SC formulation of Takeda’s Entyvio® has been approved for maintenance therapy in adults with moderately to severely active ulcerative colitis (UC) after induction therapy with IV administered Entyvio.[iii] Patients may administer their own SC dose, which may begin at or after week 6, after at least two IV administered doses. In the phase 3 VISIBLE-1 (NCT02611830) trial, after 2 doses of Entyvio IV 300 mg, significantly more patients who were switched to Entyvio SC 108 mg every 2 weeks achieved clinical remission at week 52 compared to those who were switched to placebo (46% versus 14%; p<0.001). Improvement in endoscopic mucosa appearance was also seen among patients switched to Entyvio SC (57% versus 21%; p<0.001). The Entyvio SC formulation is expected to be available as a single-dose pre-filled pen (Entyvio Pen) by the end of October 2023, with pricing to follow.
  • Pombiliti™ (cipaglucosidase alfaatga) + Opfolda™ (miglustat): The FDA approved the combination use of Pombiliti, a hydrolytic lysosomal glycogen-specific enzyme, and Opfolda, an enzyme stabilizer, for the treatment of adults with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) who weigh ≥ 40 kg and who are not improving on their current enzyme replacement therapy (ERT).[iv] Pombiliti’s weight-based dose is administered every other week as an IV infusion over 4 hours and is preceded by an oral dose of Opfolda. Pombiliti carries boxed warnings for severe hypersensitivy reactions, infusion-associated reactions, and risk of acute cardiorespiratory failure. This combination of Orphan Drugs provides a third ERT option for adults with late-onset Pompe disease, following Lumizyme® (alglucosidase alfa) and Nexviazme® (avalglucosidase alfa-ngpt), which are also approved for use in pediatric patients. FDA approval is supported by the phase 3 PROPEL study that compared Pombiliti + Opfolda to a non-U.S. approved alglucosidase alfa product.[v] At week 52, there was a numerically greater improvement in the 6-minute walk distance (6MWD) from baseline Pombiliti + Opfolda compared to alglucosidase alfa (+20.8 versus +7.2 meters, respectively; p=0.072), but it did not reach statistical superiority. Pombiliti + Opfolda did, however, lead to a nominal statistically significant improvement and clinically meaningful improvement in forced vital capacity (FVC) over alglucosidase alfa (-0.9 versus -4, respectively; p=0.023). Pombiliti and Opfolda became commercially available in the U.S. in early October 2023.
  • Bimzelx (bimekizumab-bkzx): UCB Pharmaceutical’s received FDAapproval for Bimzelx, a humanized interleukin-17A and F antagonist indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.[vi] The recommended dosage is 320 mg (as two 160 mg SC injections) self-administered every 4 weeks for the first 5 doses, then every 8 weeks thereafter. Efficacy of Bimzelx was demonstrated in four Phase 3 trials: BE VIVID (Ps-1; NCT03370133), BE READY (Ps-2; NCT03410992), BE SURE (Ps-3; NCT03412747), and BE RADIANT (NCT03536884).[vii],[viii],[ix],[x],[xi] In BE VIVID and BE READY, a significant proportion of patients treated with Bimzelx achieved an Investigator’s Global Assessment (IGA) of 0 (clear) or 1 (almost clear) (placebo-adjusted, 79% and 91%, respectively) and ≥ 90% reduction in Psoriasis Area and Severity Index (PASI 90) (placebo-adjusted, 80% and 90%, respectively). In addition, skin clearance with Bimzelx was superior to adalimumab (Humira®) in BE SURE, ustekinumab (Stelara®) in BE VIVID, and secukinumab (Cosentyx®) in BE RADIANT. Bimzelx is anticipated to be available in November 2023 with pricing to follow.[xii]
  • Zilbrysq® (zilucoplan): UCB Pharmaceutical’s complement component 5 (C5) inhibitor Zilbrysq was approved for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive.[xiii] The dosage of Zilbrysq is based on the patient’s actual body weight. Zilbrysq is the first once-daily SC self-administered complement C5 inhibitor. It Zilbrysq carries a boxed warning regarding serious meningococcal infections and is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program. In the Phase 3 RAISE trial (NCT04115293), Zilbrysq demonstrated a statistically significant improvement from baseline compared to placebo in the Myasthenia Gravis-Specific Activities of Daily Living scale (MG-ADL) total score (placebo-adjusted score, -20.9; p<0.001) at 12 weeks; a higher MG-ADL score indicates greater impairment.[xiv] In addition, 73% of patients treated with zilucoplan achieved a clinically meaningful  reduction (≥ 3-point) in the MG-ADL score without the need for rescue therapy, compared to 46% of those who received placebo (p=0.0005). It will compete with the other IV complement C5 inhibitors eculizumab (Soliris®) and ravulizumab-cwvz (Ultomiris®) FDA approved for gMG. Launch of Zilbrysqis planned by the end of 2023 with pricing to follow.
  •  Velsipity™ (etrasimod): The FDA approved Pfizer’s once daily oral sphingosine 1-phosphate (S1P) receptor modulator Velsipity for the treatment of moderately to severely active ulcerative colitis (UC) in adults.[xv] FDA approval was supported by the Phase 3 ELEVATE UC 12 (NCT03996369) and ELEVATE UC 52 (NCT03945188) clinical trials.[xvi] In ELEVATE UC 12, at 12 weeks, clinical remission of UC was achieved in significantly more patients treated with etrasimod (25%) than placebo (15%; p=0.026). ELEVATE UC 52 reported significantly higher remission rates with etrasimod compared to placebo at week 12 (27% versus 7%, respectively; p<0.0001) and at week 52 (32% versus 7%; p<0.0001). Velsipity may compete directly with once-daily, oral Zeposia® (ozanimod) in the UC setting. A head-to head trial has not been conducted and cross trial comparisons make it hard to compare the two drugs in terms of efficacy.  Like Zeposia, use of Velsipity may result in transient atrioventricular (AV) conduction delays. Use of both agents are contraindicated in patients with certain cardiovascular conditions. Velsipity is expected to be available in the in 2023 with pricing to follow.
  • Omvoh™ (mirikizumab-mrkz): Eli Lilly received FDA approval for their interleukin-23 (IL-23) antagonist Omvoh to treat adults with moderately to severely active ulcerative colitis (UC).[xvii] Treatment is started with an induction dosage of 300 mg given via IV infusion over 30 minutes every 4 weeks for 3 doses. This is followed by a maintenance dose of 200 mg (two 100 mg injections) administered SC at week 12 and every 4 weeks thereafter. The SC dose may be administered by the patient or caregiver. Mirikizumab’s action is directed against the p19 subunit of IL-23 and does not bind IL-12, which may distinguish it from the interleukin-12/23 inhibitor Stelara® (ustekinumab) that is indicated for UC.[xviii] Both agents offer induction therapy with dosing, followed by SC maintenance therapy; Stelara as a single IV induction dose followed by every 8 week SC maintenance that may be administered by the patient or caregiver. In phase 3 trials, mirikizumab led to a significantly greater rate of clinical remission compared to placebo at week 12 in the LUCENT-1 induction trial (NCT03518086) (24.2% versus 13.3%, respectively; p<0.001) and week 40 in the LUCENT-2 maintenance trial (NCT03524092) (49.9% versus 25.1%, respectively; p<0.001).[xix] Launch of Omvoh is expected within weeks of FDA approval. Pricing will follow.
  • Agamree® (vamorolone): The FDA approved Santhera’s dissociative corticosteroid Agamree for the treatment of in patients 2 years of age and older with Duchenne muscular dystrophy (DMD), a rare, genetic neuromuscular disorder that affects boys.[xx] Agamree will be supplied as an oral suspension that is dosed once daily. In the phase 2b VISION-DMD trial (NCT03439670), at 24 weeks, vamorolone 6 mg/kg/d and 2 mg/kg/d doses led to improvement over placebo in key mobility endpoints including time to stand (TTSTAND: change from baseline, +0.05 m/s [p=0.002] and +0.04 m/s [p=0.02] versus -0.01 m/s; respectively) and the 6 minute walk test (6MWT: change from baseline, +28.8 m [p=0.003] and +31 m [p=0.009] versus -23.9 m; respectively).[xxi] Corticosteroids, such as Emflaza® (deflazacort) and historically used prednisone, have been a standard of care in DMD to delay progression of muscle weakness and improve respiratory function. Agamree provides an additional oral steroid option while possibly limiting some of their negative side effects. Agamree may also be used as adjunct to antisense oligonucleotide agents (Amondys 45, Exondys 51, Vyondys 53, Viltepso®) that are approved for DMD. Launch is expected in Q1 2024, with pricing to follow.
  • Zymfentra™ (infliximab-dyyb): The FDA approved a SC formulation of Celltrion’s infliximab-dyyb, under brand name Zymfentra, for the maintenance treatment of adults with moderately to severely active UC or CD, following treatment with an IV-administered infliximab product.[xxii] Maintenance treatment with Zymfentra 120 mg SC once every 2 weeks may begin at or after week 10 of therapy.

Zymfentra was evaluated in two phase 3 trials, LIBERTY-UC (NCT04205643) and LIBERTY-CD (NCT03945019). The studies revealed at week 54, Zymfentra was superior to placebo in the primary endpoints of clinical remission of UC and CD (placebo-adjusted rates, 21% and 35%%, respectively; both p<0.0001) and endoscopic response of CD (placebo-adjusted rate, 34%; p<0.0001) as maintenance therapy after IV induction. The overall safety data were similar between Zymfentra and placebo. Zymfentra is the only infliximab product that is given via SC injection and can be administered by the patient or caregiver. Zymfentra carries a boxed warnings for serious infections and malignancy, similar to IV infliximab products, including Celltrion’s IV biosimilar Inflectra® (infliximab-dyyb). Zymfentra is not a biosimilar to Centocor’s IV-administered Remicade® (infliximab). The launch timeframe has yet to be announced. Pricing to follow launch.

New Indications

  • Enbrel® (etanercept): The FDA approved a new indication for Amgen’s Enbrel, a tumor necrosis factor (TNF) blocker, for the treatment of juvenile psoriatic arthritis in children 2 years of age and older.[xxiii]
  • Keytruda® (pembrolizumab): Keytruda, programmed death receptor-1 (PD-1)-blocking antibody by Merck, received a new indication for the treatment of patients with resectable (tumors ≥4 cm or node positive) non-small cell lung cancer (NSCLC) for use in combination with platinum-containing chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.[xxiv]
  • Opdivo® (nivolumab): The FDA expanded the melanoma indication of Bristol-Myers Squibb’s PD-1 inhibitor Opdivo for melanoma to include stage 2b and stage 2c.[xxv] The approved indication is now for the adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stages IIB, IIC, III, or IV melanoma.
  • Braftovi® (encorafenib) + Mektovi® (binimetinib): Pfizer’s kinase inhibitors Braftovi and Mektovi received a new indication for their combination use to treat adults with metastatic NSCLC with a BRAF V600E mutation, as detected by an FDA-approved test.[xxvi],[xxvii]
  • Abrilada™ (adalimumab-afzb): The FDA has designated Abrilada as an interchangeable biosimilar, for all its approved indications, to its reference product Humira® (adalimumab; Amgen).[xxviii]
  • Byooviz™ (ranibizumab-nuna): The FDA has designated Byoovix as an interchangeable biosimilar, for all its approved indications, to its reference product Lucentis® (ranibizumab).[xxix]
  • Rozlytrek (entrecitinib): The FDA expanded Genentech’s kinase inhibitor Rozlytrek solid tumor indication to include pediatric patients 1 month to 11 years of age.[xxx] The solid tumor indications include for use in patients who (1) have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, as detected by an FDA-approved test without a known acquired resistance mutation, (2) are metastatic or where surgical resection is likely to result in severe morbidity, and (3) have progressed following treatment or have no satisfactory alternative therapy. All indications for solid tumors were granted the FDA’s Accelerated Approval based on tumor response rate and durability of response. The FDA also approved a new oral 50 mg pellet formulation that may be sprinkled on soft food.
  • Idacio® (adalimumab-aacf): Fresenius Kabi gained FDA approval for Idacio, a biosimilar to Humira® (adalimumab), for the treatment of moderate to severe hidradenitis suppurativa in adults.[xxxi] Idacio carries all indications as its reference product, with the exception of Uveitis.
  • Voxzogo (vosoritide): The FDA expanded the indication of BioMarin’s C type natriuretic peptide (CNP) analog Voxzogo to increase linear growth in pediatric patients with achondroplasia with open epiphyses to include patients 4.5 months to <5 years of age based on safety and pharmacokinetic data in this population.[xxxii] Voxzogo’s achondroplasia indication was granted under an Accelerated Approval.
  • Tibsovo® (ivosidenib): After a Priority Review from the FDA, Tibsovo gained a new indication for the treatment of adults with relapsed or refractory myelodysplastic syndromes (R/R/ MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as, detected by an FDA-approved test.[xxxiii] This makes the IDH1 inhibitor by Agios the only targeted therapy approved in the U.S. to treat R/R MDS.
  • Vabysmo® (faricimab-svoa): The FDA granted approval for Genentech’s Vabysmo, a bispecific antibody, for the treatment of macular edema following retinal vein occlusion.  Vabysmo is also approved for wet age-related macular degeneration and diabetic macular edema.

New Formulation

  • Cosentyx® (secukinumab): The FDA approved an intravenous (IV) formulation of Novartis’ human interleukin-17A antagonist Cosentyx for the treatment of adults with psoriatic arthritis (PsA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA).[xxxiv] The approved 125mg/5 mL solution for IV use must be diluted prior to administration and is infused over 30 minutes per a weight-based dose. The IV formulation is not approved for pediatric indications (active enthesitis-related arthritis, AS, and PsA) nor for plaque psoriasis in adult and pediatric patients; the SC formulation is approved for these indication. The IV formulation will be available in 2023.


[i] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761354s000lbl.pdf.

[ii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf.

[iii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761133s000lbl.pdf.

[iv] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761204s000lbl.pdf.

[v] PROPEL. https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(21)00331-8/fulltext.

[vi] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761151s000lbl.pdf.

[vii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761151s000lbl.pdf.

[viii] BE RADIANT. https://www.nejm.org/doi/full/10.1056/NEJMoa2102383.

[ix] BE VIVID. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(21)00125-2/fulltext.

[x] BE READY. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(21)00126-4/fulltext.

[xi] BE SURE. https://www.nejm.org/doi/full/10.1056/NEJMoa2102388.

[xii] https://www.ucb.com/stories-media/Press-Releases/article/BIMZELXR-Approved-by-the-US-FDA-for-the-Treatment-of-Adults-with-Moderate-to-Severe-Plaque-Psoriasis

[xiii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216834s000lbl.pdf.

[xiv] RAISE. https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(23)00080-7/fulltext.

[xv] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/216956s000lbl.pdf.

[xvi] ELEVATE UC. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2823%2900061-2/fulltext.

[xvii] https://pi.lilly.com/us/omvoh-uspi.pdf?s=pi.

[xviii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761044s009lbl.pdf.

[xix] https://www.nejm.org/doi/full/10.1056/NEJMoa2207940.

[xx] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215239s000lbl.pdf.

[xxi] https://jamanetwork.com/journals/jamaneurology/fullarticle/2795868.

[xxii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125544s018lbl.pdf.

[xxiii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/103795s5595lbl.pdf.

[xxiv] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/125514s139lbl.pdf.

[xxv] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/125554Orig1s121lbl.pdf.

[xxvi] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/210496s015lbl.pdf.

[xxvii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/210498s009lbl.pdf.

[xxviii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761118s007lbl.pdf.

[xxix] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761202s006lbl.pdf.

[xxx] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/212725Orig1s009lbl.pdf.

[xxxi] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761255s002lbl.pdf.

[xxxii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/214938s002lbl.pdf.

[xxxiii] https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/211192s000lbl.pdf.

[xxxiv] https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761349s000lbl.pdf.

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